Canadian Stem Cell Foundation Blog

Unveiling its 2016 funding awards last week, the Stem Cell Network announced support for six clinical research trials for new cell-based treatments.…

Unveiling its 2016 funding awards last week, the Stem Cell Network announced support for six clinical research trials for new cell-based treatments.

“The regenerative medicine research sector is fueled by stem cells and today it is at a tipping point, with the potential to see breakthroughs in our generation,” said Dr. Michael Rudnicki, Scientific Director of the Stem Cell Network.

One of the big winners was Dr. Lauralyn McIntyre, a researcher/clinician at The Ottawa Hospital and a professor at the University of Ottawa. Her team receives $1 million of the $9 million in announced funding to conduct a cross-Canada Phase 2 clinical trial of mesenchymal stem cell therapy for septic shock. We profiled her work on the deadly condition here.

Her colleague at The Ottawa Hospital, Dr. Duncan Stewart, will use his $999,546 award to advance his work testing enhanced angiogenic cell therapy for acute heart attacks. Check out Dr. Stewart’s Q&A here.

As well, Dr. Sandra Cohen at the Hôpital Maisonneuve Rosemont in Montreal will investigate ways to improve the expansion of cord blood hematopoietic stem cells via her $999,968 award.

Two diabetes trials were also funded: Dr. Timothy Kieffer of the University of British Columbia gets $500,000 to test a stem cell therapy for insulin replacement, while Dr. James Shapiro of the University of Alberta receives almost the same amount for a clinical trial to solve the “supply and survival problem” in using stem cell transplants. Both Dr. Kieffer and Shapiro also receive $500,000 in funding through the Network’s Disease Team Program.

In all, 31 projects from across Canada will receive funding to help move research from lab bench to bedside in areas such as brain injury, kidney disease and breast cancer.

Making the announcement, Science Minister Kirsty Duncan said the investment will help translate discoveries into better health and economic growth for Canadians. It was made possible with the announcement of a two-year, $12-million extension of the Stem Cell Network in the March federal budget.

In a news release, Dr. Stewart said the funding “brings us a big step closer to figuring out how to harness the incredible potential of stem cells to treat devastating diseases.”

In the summer of 2014, Dan Muscat’s fingertips started to tingle. It felt like tiny shards of glass were piercing the skin underneath his fingernails.…

In the summer of 2014, Dan Muscat’s fingertips started to tingle. It felt like tiny shards of glass were piercing the skin underneath his fingernails. Over the next weeks and months the pain crept into his hands.

His family doctor thought it might be arthritis and prescribed a steroid to get things under control. “Within days, the disease sped up,” says Muscat. “It went from the top of my head to my toes. My ankles and my feet were so inflamed I couldn’t walk 20 feet.”

Muscat, who owns a jewelry business in St. Thomas, Ontario, was referred to a rheumatologist who immediately recognized what was happening to him: “Dan had rapidly progressive early onset diffuse scleroderma,” says Dr. Janet Pope of St. Joseph’s Health Care in London.

Scleroderma — from the Greek words for “hard” (sclera) and “skin” (derma) — is the term used for a group of rare autoimmune diseases that, according to the Mayo Clinic, involve hardening of the skin and connective tissues due to an overproduction of collagen. Diffuse scleroderma is a systemic type of the disease, meaning it affects not only the skin and muscle but also the digestive tract and internal organs, ultimately leading to death.

The future did not look good for Muscat: “He had not responded to usual immune suppressive treatment,” says Dr. Pope.

By late 2015, Muscat was growing desperate. He had heard that Dr. Richard Burt at Northwestern University in Chicago was offering a treatment using a patient’s own bone marrow stem cells to rebuild their immune system and shut down scleroderma. Muscat reached out to the Canadian Stem Cell Foundation for guidance.

“I investigated costs of Northwestern University treatment,” says Muscat, “and it was somewhere between $300,000 and $350,000. I thought about selling my business or my home. If I had to, I would have done that. Financially, it would have been a huge burden.”

Because the American treatment is similar to the combination chemotherapy/ bone marrow stem cell transplant treatment that The Ottawa Hospital’s Dr. Harry Atkins has used successfully against extreme forms of multiple sclerosis (MS, which is also an autoimmune disorder), the Foundation suggested Muscat reach out to him. Earlier this year Dr. Atkins agreed to take Muscat on as his first scleroderma patient.

“He fit the profile of the kind of patient best treated with these kinds of stem cell transplants” says Dr. Atkins, who is also an associate professor at the University of Ottawa. “There was a randomized trial carried out in Europe that was very well done and had very good results. I know the doctors involved and we’d talked a little bit about it, but their methods had also been published and are available for everybody. That’s why we publish — to spread the knowledge.”

By the time Muscat arrived in Ottawa in May, the disease had taken its toll. Once an imposing figure who worked out regularly and thought nothing of going for five- or six- miles runs, Muscat had watched 45 pounds fade from his six-foot frame. His brisk walk had turned into a painful shuffle. His boundless energy (he built a log home by himself, is an avid fisherman and collects classic sports cars) disappeared, making it difficult to get through a day. His fingers became like stone claws, curving in at the knuckles. His skin was so impenetrable he worried the Ottawa medical team might not be able to run an intravenous line. And the pain was excruciating.

“On a scale of 1 to 10, about 15,” Muscat says.

Muscat began the therapy in June and spent much of last summer undergoing the treatment designed to destroy the diseased immune system and rebuild it with his own purified stem cells, ideally to produce a new, scleroderma-free one. Though not as harsh as the chemotherapy MS patients undergo, it is not a treatment to be taken lightly. The risk did not dissuade Muscat: “If the treatment didn’t kill me, the disease would.”

He made it through chemo with few noticeable side-effects beyond losing his hair and tolerating a couple of infections. Released from hospital in August, he embraced physiotherapy, doing three to five hours daily to get his flexibility back and rid his body of the collagen buildup.

He is now back in St. Thomas, occasionally working at the store — he has to limit his time in public places because the chemo has left him vulnerable to infections. “My pain is one or two percent compared to what it was before.” His range of motion with his arms and legs is much improved and he can get around on foot again.

He realizes it could take a year before the chemo drugs fade from his system and the full benefits of the treatment kick in. Muscat, grateful for the excellent care provided by The Ottawa Hospital’s bone marrow transplant team, is unswerving in his belief that he can beat this disease. “I feel physically and mentally better. I just don’t feel that I’m being attacked.”

In an email message, rheumatologist Dr. Pope said that while Muscat “has a long road ahead, we expect more recovery with softening of his skin and improved function. His pain is less and he feels that shortly after the transplant a ‘switch’ was turned back to how it should be and he developed a better sense of well-being. I will follow him carefully and I hope for more improvement over the next year or two. He is in physiotherapy and is getting ongoing intravenous drugs for safety as his immune system continues to reconstitute. I am very hopeful that Dan will be even better over time. This is not a cure, but has given Dan a better chance of recovery.”

In Ottawa, Dr. Atkins also urges caution. “It’s going to take months and months to know if this is a real improvement. The changes with scleroderma don’t happen overnight. This is not a curative treatment; it doesn’t make scleroderma go away and never come back. It’s something that helps a population of scleroderma patients: some patients improve, some not. There are patients that progress or relapse again.”

Muscat is positive about the future: “I still have the remnants of the disease, but I can jump in and out of my truck again and I can walk a couple of kilometres with no problems. I feel very optimistic, like I’ve been given a second chance.”

We can add another disease to list of conditions that can now be treated with bone marrow stem cell transplants: myasthenia gravis (MG).…

We can add another disease to list of conditions that can now be treated with bone marrow stem cell transplants: myasthenia gravis (MG).

This week, CTV News in Ottawa told the story of Anne Scott, a 58-year-old Kemptville, Ontario woman whose MG refused to respond to conventional therapies.

MG is an autoimmune disorder like multiple sclerosis (MS). It interrupts communication between the muscles and the nerves, which can make swallowing and breathing difficult.

While most patients can be treated with conventional therapies, some, like Scott, cannot. She had to be placed on life support a dozen times.

Under the supervision of The Ottawa Hospital’s Dr. Harry Atkins, Scott underwent a chemotherapy/stem cell procedure. Essentially, it involves one using a patient’s own blood stem cells to rebuild their immune system after it has been wiped out by chemo therapy. If all goes well, the rebuilt immune system is free of the disease.

Dr. Atkins has had success using this approach to treat patients with MS, Stiff Person’s Syndrome, neuromyelitis optica and Crohn’s disease. The procedure has been used to treat leukemia and other forms of blood-based cancer for decades.

Scott, who was able to attend her daughter’s wedding shortly after undergoing the procedure, was one of seven patients in a study led by Dr. Atkins, the results of which have been reported in in JAMA Neurology All seven patients no longer need treatment for MG and are symptom-free.

Dr. Paola Marignani is searching for new treatments. More precisely, the Dalhousie Medical School researcher wants a new combination of drugs to target cancer in a new and different way.…

Dr. Paola Marignani is searching for new treatments. More precisely, the Dalhousie Medical School researcher wants a new combination of drugs to target cancer in a new and different way.

“Many of the drugs we have in the clinic today block oncogenes that were discovered 15 or 20 years ago,” says Dr. Marignani, pictured at right. “We can’t just keep using the same drugs over and over again in different combinations. We need to find new targets and new drugs.”

Her key target is LKB1, a protein that has multiple functions throughout the body, including tumour suppression. LKB1 is often found to be missing or mutated in breast cancer, lung cancer, pancreatic cancer and other forms of the disease. “If you lose LKB1 or its mutated, it sets up protein signalling pathways for disaster,” says Dr. Marignani. “It would be like an intersection that once had stop signs and is now without any. Eventually an accident is going to happen.”

To understand what happens when LKB1 is lost and mutated, her team used re-engineered mice without the ability to express the protein. That led them to finding a combination of compounds that shuts down aggressive, metabolically active HER2-positive breast cancers — a common form of the disease — in the rodents.

Dr. Marignani is using a two-pronged approach by testing the new compounds in combination with Herceptin®, which has become the standard of care for HER2-positive breast cancer. “We know Herceptin® is effective,” says Dr. Marignani. “We can use the discovery we made in mice to strategically attack the cancers from multiple branches using new drugs in combination with the standard treatment of care, and see what happens.”

So far, she is cautiously optimistic. “The animals tolerate the drug combination, which is very important, and early data suggests the tumours are not progressing.”

Beyond shutting down tumour growth, Dr. Marignani and her team want to find ways to stop cancer from recurring by killing off the cancer stem cells that resist the original treatment.

“There is always the possibility that there are some cancer stem cells hiding out, just waiting, that have developed resistance to the treatment that killed off the bulk of the cancer,” she explains. “We have seen that our drug combination reduced the proteins that drive recurrence. We did not anticipate this would happen because there was no evidence in the literature. In our current study, we need to consider whether stem cells play a role in cancer recurrence in our model and look for pathways that are active in those cells. We don’t know yet. We’re working on it.”

With fine tuning of her animal-model preclinical work done, Dr. Marignani hopes to move into a Phase 1 clinical trial, testing the safety of the drug combination in humans. But she emphasizes that the work is in the early stages.

“It is important that we look for new treatment possibilities even though the current treatments are reasonably good. Targeting oncogenes has served us well, however it is time we expand our toolbox. We can do better.”

After a decade of innovation, McMaster’s Stem Cell and Cancer Research Institute (SCC-RI) has launched a new website to draw attention to research advances and share links to resources for patients, care givers and future scientists. …

After a decade of innovation, McMaster’s Stem Cell and Cancer Research Institute (SCC-RI) has launched a new website to draw attention to research advances and share links to resources for patients, care givers and future scientists. The SCC-RI’s new logo, a stylized hand, represents the numerous people involved in translating research into new therapeutic options for patients.

The SCC-RI was established in 2006 — the same year Dr. Shinya Yamanaka established his game-changing protocol to turn adult skin stem cells back to an embryonic stem cell-like state of pluripotency — to drive new therapies to the clinic. Since the beginning, SCC-RI has focused its research on improving bone marrow and cord blood transplants, finding cell-based solutions to cancer and identifying and targeting the cells responsible for neural disorders such as autism.

“Our commitment to working with human cells and our established drug discovery capabilities make this the best place for moving forward to patient-specific drug discovery,” said Dr. Karun Singh, Principal Investigator at SCC-RI in a recent blog post.

Dr. Singh recently led a team that discovered a gene mutation that causes autism spectrum disorders (ASD). Having identified the genetic glitch, researchers can now focus their efforts on finding a way to improve the brain connections that are causing symptoms of ASD.

The SCC-RI team has developed a robotic system to test a library of currently available drugs on a variety of diseased cells before starting human trials. In a landmark study, Dr. Mick Bhatia, SCC-RI Director and Senior Scientist, found the antipsychotic drug, thioridazine, kills cancer stem cells responsible for initiating leukemia without harming normal stem cells. A Phase I clinical trial is now underway.

Dr. Allen Eaves, President and CEO of STEMCELL Technologies Inc., has been awarded Entrepreneur of the Year Pacific Winner by Ernst and Young Canada.…

Dr. Allen Eaves, President and CEO of STEMCELL Technologies Inc., has been awarded Entrepreneur of the Year Pacific Winner by Ernst and Young Canada.

The theme for the 2016 awards is recognizing accelerators — those entrepreneurs who “accelerate all of us by bringing us new products and services and by driving our economy” explains Lui Pettrollini, Entrepreneur of the Year Pacific Program Director, in an article carried in the Globe & Mail’s Report On Business Magazine.

Dr. Eaves founded STEMCELL in 1993 to support his own research as the head of the BC Cancer Agency’s Terry Fox Laboratory in Vancouver. Unable to source what he needed to support his research, Dr. Eaves began making media for growing blood-forming stem cells. Today, STEMCELL Technologies offers more than 2,000 cell biology tools to researchers in over 80 countries. It is Canada’s largest biotech company with 850 employees and $150 million in annual sales.

“I run my company like a graduate training program,” Dr. Eaves said in the ROB Magazine piece. “Scientists have a lot of freedom in deciding what they can work on and that inspires them to be creative and think clever thoughts.”

Dr. Eaves has also developed a group of companies including STEMSOFT Software Inc., that makes software for managing data in bone marrow centres, cord blood banks, cellular therapy companies as well as tumour and tissue repositories. He also founded Malachite Management Inc., which provides association management, conference management, fundraising and strategic planning to medical and scientific organizations.

Dr. Eaves is a major supporter of the Canadian Stem Cell Foundation as both a member of the Board of Directors and a founding Till & McCulloch Leadership Circle member.

Dr. Eaves’ accomplishments will be celebrated along with other regional winners on November 22nd at the EY National Awards Gala in Toronto.

A feature in the current edition of Biotechnology Focus tells the story of how systemic scleroderma was stealing the life of Dan Muscat, a 49-year-old St.…

A feature in the current edition of Biotechnology Focus tells the story of how systemic scleroderma was stealing the life of Dan Muscat, a 49-year-old St. Thomas, Ontario jeweler. After a bone marrow stem cell treatment at The Ottawa Hospital last summer, his excruciating pain has diminished and he has been able to resume much of his active life. Though not cured, he has a new lease on life.

Dan Muscat’s story raises a key question: As stem cells begin to emerge as treatments for a number of currently incurable conditions and diseases, what will it take to get more therapies in the clinic?

The article quotes James Price, President & CEO of the Canadian Stem Cell Foundation: “To make this happen, we need to think differently– and to think big.” He points out that a coordinated national stem cell strategy would not only lead to cures and save lives, but also generate thousands of good jobs and boost the economy.”

Japanese researchers have found that a genetic mutation linked to schizophrenia changes the way brain cells develop and differentiate.

According to a report in today’s Guardian newspaper, the alteration of cell development in the brain changes the normal balance of neurons (nerve cells) and connective tissue in the brain (glia).…

Japanese researchers have found that a genetic mutation linked to schizophrenia changes the way brain cells develop and differentiate.

According to a report in today’s Guardian newspaper, the alteration of cell development in the brain changes the normal balance of neurons (nerve cells) and connective tissue in the brain (glia).

The researchers’ findings, published in Translational Psychiatry, suggest that abnormal neural differentiation leads to fewer neurons and more non-neuronal cells being produced during early stages of brain development, which could be contributing to the presence of the disease.

Dr. Manabu Toyoshima of Japan’s RIKEN Brain Science Institute extracted skin cells from two female schizophrenic patients and two healthy individuals, then reprogrammed them to generate induced pluripotent stem cells (iPS cells), which are like embryonic stem cells in that they have the capacity to differentiate into any cell type in the body.

In essence, the scientists found that the neurospheres (clusters of neural stem cells) derived from the IPS cells of the schizophrenic patients were smaller and produced fewer neurons but significantly more astrocytes — star shaped glial cells.

The findings could help scientists better understand the origins of schizophrenia, a common form of mental illness that affects about one in 100 people and, as the Guardian points out, “is known to be highly heritable, but is genetically complex.”

Fifty-five years after they became the first in the world to identify stem cells, Drs. James Till and Ernest McCulloch are now a must-see attraction at the Telus World of Science in Vancouver.

Artist Ruth Abernethy’s “portrait in bronze” of the two Canadian researchers was unveiled yesterday outside the main doors of the science centre. Within minutes, children were climbing the stools of the interactive artwork and clamouring to have their photos taken with the great men.

The work of art was commissioned by Dr. Allen Eaves, founder and CEO of STEMCELL Technologies Inc., Canada’s leading biotech company, to honour Till & McCulloch who are well known within the field as the Fathers of Stem Cell Science but virtually unknown to most Canadians. Dr. Eaves and his wife Dr. Connie Eaves, an internationally respected stem cell researcher, worked with Drs. Till and McCulloch decades ago at the Ontario Cancer Institute in Toronto.

As Science World CEO Scott Sampson said in his address to the crowd that gathered at Science World, the tribute is a reflection of new-found Canadian pride in our country’s outstanding achievements in a number of fields.

Billed as “Canada’s premier stem cell research event” the Till & McCulloch Meetings provide an excellent opportunity for young scientists to discuss their work with sector leaders and talk about how they can play a role in advancing regenerative medicine.…

Billed as “Canada’s premier stem cell research event” the Till & McCulloch Meetings provide an excellent opportunity for young scientists to discuss their work with sector leaders and talk about how they can play a role in advancing regenerative medicine.

Named after Drs. James Till and Ernest McCulloch, who discovered stem cells in 1961, the annual conference attracts hundreds of participants from across Canada and all over the world. The meetings, which begin Monday and run through Wednesday in Whistler, BC, foster new collaborations and nurture Canadian leadership and innovation.

Since the conference was first organized in 2001, much effort has been made to support young scientists via networking sessions, workshops and presentations. Top trainees are acknowledged with awards for their poster submissions, presentations and research efforts

Recognizing the importance of the trainee experience in Canada, a special session will be held to develop programs for young investigators. Participants will be asked to recommend the types of workshops and skills development best suited to support trainees and will participate in organizing them. Previous sessions have included workshops in career development, grant review and journal writing and ethics.

For more information on the Till & McCulloch Meetings 2016 click here.

Fifty-five years ago, Drs. James Till and Ernest McCulloch identified stem cells and provided the theoretical underpinning for bone marrow transplant procedures that have saved the lives of countless leukemia patients.…

Fifty-five years ago, Drs. James Till and Ernest McCulloch identified stem cells and provided the theoretical underpinning for bone marrow transplant procedures that have saved the lives of countless leukemia patients. It also opened up the field of stem cell science.

To commemorate the breakthrough, which ranks as one of Canada’s greatest medical discoveries, Science World at TELUS World of Science in Vancouver will unveil a bronze portrait of Drs. Till and McCulloch on Sunday.

“It’s impossible to overstate the impact of Dr. Till and Dr. McCulloch’s discovery and their long-time collaboration,” says, Dr. Allen Eaves, President & CEO of STEMCELL Technologies Inc. that commissioned the work of art. “Their work changed the course of cancer research and paved the way for what we now call regenerative medicine.”

Both Dr. Eaves, who co-founded the Terry Fox Laboratory with the BC Cancer Agency, and his wife, prominent cancer researcher Dr. Connie Eaves, were greatly influenced by Drs. Till and McCulloch during their time at the Ontario Cancer Institute in Toronto. Connie was a post-doctoral fellow who worked closely with them and Allen used their methodology in his own cancer research, which led him to set up the first bone marrow transplantation program in Western Canada.

The sculpture was created by renowned artist Ruth Abernethy, whose public portraits in bronze have celebrated the achievements of several prominent Canadians. She is probably best known for her depiction of Glenn Gould sitting on a bench at CBC in Toronto and Oscar Peterson tinkling a piano outside the National Arts Centre in Ottawa. She will attend the unveiling Sunday to talk about her work and sign copies of Life and Bronze: a Sculptor’s Journal.

The accomplishments of Drs. Till and McCulloch are also celebrated in the book Dreams & Due Diligence: Till and McCulloch’s Stem Cell Discovery and Legacy. Author Joe Sornberger will be at the unveiling Sunday to sign copies of his book. The book is available for purchase on our website by clicking on this link.

The public is welcome to attend the unveiling at Science World at 1455 Quebec Street on Sunday at 1 p.m. Next spring, a sister bronze portrait will be installed at the MaRS Building in downtown Toronto.

Suppose you have a disease for which there currently is no cure. You go online and find a stem cell clinic in the United States offering to rid you of the condition with their miraculous new treatment. …

Suppose you have a disease for which there currently is no cure. You go online and find a stem cell clinic in the United States offering to rid you of the condition with their miraculous new treatment. The clinic looks reputable enough and even has certified doctors on staff. When you investigate further you find out that it will set you back somewhere between $15,000 and $25,000. What should you do?

Dr. Lawrence’s made the comments in an Orange Counter Register news feature this week that warns people “whose lives – or whose loved ones’ lives – have been upended by chronic or incurable conditions such as Parkinson’s” to be cautious about clinics offering unproven stem cell cure-alls.

“If somebody is telling you something that’s too good to be true, it’s too good to be true,” says Dr. Sidney Golub, director of the Sue and Bill Gross Stem Cell Research Center at UC Irvine.

We have frequently used this blog to urge caution to those considering stem cell treatments abroad, especially in countries with less-stringent health regulations and clinics offering to treat a laundry list of diseases and conditions with the same stem cell “cure.”

But as the Register piece indicates, there are now nearly 600 clinics in the United States — just a quick drive or flight away for most Canadians — touting expensive stem cell treatments for everything from breast augmentation to spinal cord injuries. Many use stem cells drawn from the patient’s own fat, arguing that such treatments don’t require approval from the U.S. Food and Drug Administration (FDA).

Our website has a section on stem cell tourism. You can find it here. It links to an excellent guidebook produced by the University of Alberta’s Faculty of Law, Albany Medical College and the Stem Cell Network. There is also a great web resource provided by the International Society for Stem Cell Research. You can find it here.

Beyond the possibility of spending money on a treatment that does not work, there is always a risk that the procedure may have adverse side effects. “The risk is far greater than the potential benefit,” Mary Bass, a director of the patient advocacy group Americans for Cures, told the Register.

The FDA also warns that patients may be “vulnerable to unscrupulous providers of stem cell treatments that are illegal and potentially harmful.”

Two Federal Government departments — Innovation, Science and Economic Development Canada and Health Canada — have asked the Council of Canadian Academies (CCA) to study and report back on the state of regenerative medicine in Canada.…

Two Federal Government departments — Innovation, Science and Economic Development Canada and Health Canada — have asked the Council of Canadian Academies (CCA) to study and report back on the state of regenerative medicine in Canada.

“We are very pleased to receive this request from the Government,” says Dr. Eric M. Meslin, President and CEO of the CCA, in a media release. “Canada is a global leader in regenerative medicine and we look forward to contributing to the evidence base for making decisions about how this country can continue to excel in this incredibly important field of medicine.”

The council will conduct an “expert panel workshop” to address two key questions:

What are Canada’s strengths in regenerative medicine (and why are they strengths)?

What are the opportunities that exist and barriers that must be overcome for Canada to ensure that it can excel in regenerative medicine in the international arena?

“This is good news for advancing stem cell R&D,” says James Price President & CEO of the Canadian Stem Cell Foundation, adding that the move shows that regenerative medicine is a priority for the Government.

“As the champion of the Canadian Stem Cell Strategy, which would see more stem cell research leap the hurdle to clinical trials and new treatments, we applaud the Government for commissioning the workshop. We look forward to helping the CCA in any way possible.”

The CCA, a not-for-profit organization that undertakes independent, authoritative, evidence-based assessments to inform public policy, is assembling a multidisciplinary, multi-sectoral, group of experts to participate in the two-day workshop expected to take place in the coming weeks. The results will be published in early 2017.

Back in February, we reported on how thousands of people are visiting hundreds of clinics in American cities and some Canadian centres to get an expensive, unproven, same-day stem cell treatment to ease pain and restore flexibility in their arthritic or banged-up knees.…

Back in February, we reported on how thousands of people are visiting hundreds of clinics in American cities and some Canadian centres to get an expensive, unproven, same-day stem cell treatment to ease pain and restore flexibility in their arthritic or banged-up knees.

The BMAC (bone marrow aspirate concentrate) procedure involves extracting mesenchymal stem cells from a patient’s pelvis, spinning them in a centrifuge and then re-injecting them into the damaged knees. The treatment can cost anywhere from $2,000 to $10,000 per injection, even though there are no published reports from high-level clinical trials to indicate it works.

We are, however, getting closer to knowing the truth.

Dr. Shane Shapiro, an orthopedic surgeon at the Mayo Center for Regenerative Medicine in Jacksonville, Florida, has published the results of a 25-person clinical trial in which patients with osteoarthritis received an injection of stem cells in one knee and a saline solution placebo in the other — unaware of which was which.

The results are less than conclusive. As reported in the American Journal of Sports Medicine:

“Study patients experienced a similar relief of pain in both BMAC- and saline-treated arthritic knees. Further study is required to determine the mechanisms of action, duration of efficacy, optimal frequency of treatments, and regenerative potential.”

According to Dr. Shapiro, this leads to two possible interpretations. One is that the BMAC procedure was no better than placebo (because both knees got equally better). The second is that stem cells have a systemic effect (which some animal and human studies suggest) and can go to where needed — so that stem cells injected into the left knee could also make the right knee feel better.

Dr. Shapiro stresses that his study doesn’t prove the latter. It’s still just a theory.

Clearly, what’s needed is a double-blind, randomized controlled clinical trial in which each participant is assigned to receive either the BMAC treatment or a placebo by chance with neither the patient nor the doctor knowing who gets which so that the results can be objectively compared. Until then, we won’t know whether the BMAC procedure actually relieves pain and repair knees.

It should also be noted that the Mayo clinic used the most promising stem cells for their injections. Instead of just drawing and concentrating bone marrow and then reinjecting it into the patient, Dr. Shapiro’s team tracked the number of stem cells and tested their viability before injecting them into a patient’s knees. A patiently likely would not get that level of quality assurance from a busy clinic trying to treat as many patients as quickly as possible.

When it comes to innovation, Canada’s stem cell community is ready to roll.

That’s the message conveyed in an opinion piece in iPolitics, the must-read publication for those involved in — or merely fascinated by — Canadian politics.…

When it comes to innovation, Canada’s stem cell community is ready to roll.

That’s the message conveyed in an opinion piece in iPolitics, the must-read publication for those involved in — or merely fascinated by — Canadian politics.

The article responds to the call from Navdeep Bains, Canada’s Minister of Innovation, Science and Economic Development, for “fresh ideas and a joint action plan that will make innovation a national priority and put Canada on a firm path to long-term economic growth.”

The iPolitics piece points out that Canada’s stem cell community has “seen the writing on the wall, consulted, and forged a plan to act. It’s a blueprint designed to ensure our world-class talent grows here at home, that Canada reaps the economic rewards and that we remain a global leader in this sector. The Canadian Stem Cell Strategy has tremendous support across the country and is ready to be implemented.”

Three powerful voices united to co-author the article:

Peter MacKinnon, LLD, is President Emeritus of Athabasca University and former President and Vice-Chancellor of the University of Saskatchewan. His national appointments include the Prime Minister’s Advisory Committee on the Public Service and the Science, Technology and Innovation Council of Canada

Molly Shoichet, PhD,holds the Tier 1 Canada Research Chair in Tissue Engineering at the University of Toronto. She was the North American recipient of the L’Oréal-UNESCO Awards for Women in Science in 2015

James Price, MBA,is President & CEO of the Canadian Stem Cell Foundation.

“We wanted to make it clear that by making the Strategy a component of its Innovation Agenda, the Government will be putting stem cells to work for Canada’s future,” said Mr. Price. “Not only will this set things in motion for many more people to be treated with new therapies, it will boost the economy and assure that thousands of new high-quality jobs will be created in this burgeoning industry.”

Jonathan Pitre, Ottawa’s much-loved “Butterfly Boy,” has had to cope with daily pain that few of us could endure for his entire 16 years.…

Jonathan Pitre, Ottawa’s much-loved “Butterfly Boy,” has had to cope with daily pain that few of us could endure for his entire 16 years.

And while there is considerable hardship ahead as he waits to see whether his stem cell transplant takes, at least now there is hope.

Diagnosed shortly after birth with severe form of epidermolysis bullosa (EB), the term used for a group of diseases that cause the skin to break out in sores and blisters, Jonathan recently underwent nine days of chemotherapy at the University of Minnesota Masonic Children’s Hospital in Minneapolis before receiving a bone marrow stem cell transplant last Thursday. That was followed by second round of chemotherapy to prevent complications from graft-versus-host disease.

Now he waits to see if the transplant triggers his body to begin producing new cells that can give his ravaged skin the chance to heal.

“The next three or four weeks are going to be a critical part of Jonathan’s treatment,” explains Andrew Duffy, who has told the young man’s courageous story so sensitively in the pages of the Ottawa Citizen, his words accompanied by powerful video and photographic images by Julie Oliver.

While there is no talk of curing Jonathan, stem cells are his best hope. The physician who is pioneering this treatment, Dr. Jakub Tolar, told the Citizen that the procedure comes with the possibility of “hundreds of complications” and that he “cannot promise success.” However, in the best case scenario “the change is profound.”

Tina Boileau — Jonathan’s mother, constant caregiver and stem cell donor — has a simpler goal in mind for her son: “A day without pain.”

Kids with EB are called “Butterfly Children” because their skin seems as fragile as that of a butterfly. There is currently no cure for EB. According to the U.S. Mayo Clinic, many patients with severe forms of EB die in childhood. Others often succumb to skin cancer in young adulthood.

Across Canada, people are pulling for the remarkable young man who, throughout his battles with EB, has never lost his sense of humour, or his commitment to persevere.

An avid hockey fan, Jonathan drew national media attention when he was hired for one day to scout a game for the Ottawa Senators. He’s also the subject of a compelling TSN documentary in which he explained why he chose to become an ambassador for children like him who struggle with the disease and to campaign to raise money for research: “I wanted to start helping other people with EB.”

Even while suffering the nasty after-effects of chemo, Jonathan remains upbeat. “We’re still here, obviously, and we’re still fighting — and that won’t change,” he told the Citizen on Monday.

(This post is one of several addressing a single subject today in a blog carnival to mark the 10th anniversary of the discovery of induced pluripotent stem cells.…

(This post is one of several addressing a single subject today in a blog carnival to mark the 10th anniversary of the discovery of induced pluripotent stem cells. Please click here to read what other bloggers have to say.)

As summer holidays wind down, most parents are now too familiar with the following question: Are we there yet?

Any family trip to a campground, cottage or Nana’s house in Northport starts off with excitement for all concerned, what with getting up early, packing the car and hitting the road. For kids, though, it’s magical. Then, after about an hour of travelling, regardless what onboard entertainment you’ve arranged, boredom sets in. Two hours into the trip, they’re sure you’re never going to get them there.

Waiting for stem cell discoveries to turn into actual treatments is a lot like that, except that instead of hours, it’s decades. Instead of frustration, the feeling is desperation.

Consider the remarkable discovery, by Japan’s Dr. Shinya Yamanaka, that adult cells extracted from skin can be reprogrammed to an embryonic stem-cell-like state to reproduce any cell required for transplant or to repair organs and tissue. We first heard about these induced pluripotent stem cells (iPS cells) on August 25, 2006. A decade later, we’re still waiting for the Nobel Prize-winning work to turn into treatments.

People, especially those suffering from life-threatening diseases, want to know why we’re still waiting. Unlike bored children, they have far more riding on the answer to the Are we there yet? question. For them, it’s life and death.

At the Canadian Stem Cell Foundation hardly a day goes by that someone doesn’t contact us seeking a stem cell treatment for themselves or a loved one. Today, it was a 48-year-old Toronto man whose doctor had told him his ALS will kill him. At least we could point him in the direction of Dr. Eva Feldman at the University of Michigan, who is trying to get a Phase 2 clinical trial going on a stem cell treatment for ALS.

In all areas of medical research, the wheel turns slowly. It can take decades of lab work and clinical trials and hundreds of millions of dollars to bring new treatments to patients. Consider cervical cancer, the second most common cancer among women. In 1972, Germany’s Dr. Harald zur Hausen started working on the notion that the disease is caused by a virus. It took about 35 years — most of his career — for HPV vaccines to make it to market.

It’s even more complicated for stem cells, a relative newcomer to the scene. While their existence was proven in Canada by Drs. James Till and Ernest McCulloch in the early 1960s, the focus afterwards was on bone marrow stem cells for treating leukemia. Embryonic stem cells have only been in play since 1998. Realistically, iPS cells are still the new cells on the block.

Cell-based therapies represent a whole new way of thinking about treating diseases, and regenerative medicine is a disruptive technology. Unlike a vaccine or a drug, the actual therapy isn’t an easy thing to grasp for industry, whose commitment is crucial in moving things from the lab bench to the hospital bed.

What’s needed, then, is an innovative approach. We need to think bigger. That’s why our Foundation is championing the Canadian Stem Cell Strategy, a private/public partnership to deliver up to 10 new curative therapies to the clinic within 10 years. Crafted in consultation with 150 scientists, doctors, leaders from health charities, industry experts and philanthropists, it is backed by an in-depth KPMG study and endorsed by an international panel of experts.

The private sector is already at the table, pledging more than $350 million toward R&D — almost one-quarter of the $1.5-billion Strategy. Other industry partners, health charities and leading Canadian philanthropists are prepared to make major contributions upon demonstration of a federal commitment to the plan. We’re asking the Government of Canada, as part of its Innovation Agenda, to provide one-third, about $50 million annually over 10 years.

Are we there yet? Clearly not. But a coordinated national road map can get us there.

Congratulations to Dr. Michael Rudnicki at The Ottawa Hospital who has been awarded $4.9 million to further his research into stem cells and muscle regeneration.…

Congratulations to Dr. Michael Rudnicki at The Ottawa Hospital who has been awarded $4.9 million to further his research into stem cells and muscle regeneration.

Dr. Rudnicki, who is also Scientific Director of the Stem Cell Network and a University of Ottawa professor, received the grant from the Canadian Institutes of Health Research (CIHR) as part of its Foundation funding competition to provide the country’s top health researchers with stable, long-term support.

His colleague Dr. Lynn Megeney also received funding, under CIHR’s Project competition, to study heart muscle regeneration and remodeling.

Dr. Rudnicki is one of the world’s leading researchers in stem cells and muscle regeneration. Last fall, his lab published a paper in Nature Medicine that could completely alter perceptions on how Duchenne muscular dystrophy happens — linking it to intrinsic defects in the function of muscle stem cells

Summer isn’t really summer without a local fair or carnival to enjoy — a place you can have a variety of experiences, from riding bumper cars and tasting weird food treats to seeing expertly executed home crafts.…

Summer isn’t really summer without a local fair or carnival to enjoy — a place you can have a variety of experiences, from riding bumper cars and tasting weird food treats to seeing expertly executed home crafts.

Next week we will be adopting the carnival concept to discussing stem cells. Organized by Stacey Johnson at the Canadian Centre for the Commercialization of Regenerative Medicine (CCRM), the Thursday, Aug. 25 blog carnival will feature blogs from across the stem cell research community — including this one — posting different perspectives on a single subject. Here’s what everyone will be blogging about:

It’s been 10 years since scientists Shinya Yamanaka and Kazutoshi Takahashi announced their discovery on Aug. 25, 2006 that adult cells could be reprogrammed into induced pluripotent stem (iPS) cells to look and function like embryonic stem cells. In the decade since their discovery, converting this science into useful treatments for diseases like heart failure or diabetes has yet to be fully realized. When do you think this technology will result in commercial products or new revolutionary medical treatments, and why?

It all happens in one week’s time. You can find out more about the blog carnival at CCRM’s highly informative Signals blog. Signals will be the site where, on Aug. 25, you can link to other writers’ unique insights on the subject. Watch for it!

You read about a stem cell breakthrough that could lead to a whole new way of treating — maybe even curing — a disease. …

You read about a stem cell breakthrough that could lead to a whole new way of treating — maybe even curing — a disease. Then … nothing happens.

The gap in time between a medical science discovery and actual clinical application can seem like a millennium, especially if you are living with the disease in question.

This week in the Toronto Star, Dr. Molly Shoichet articulately — and compassionately — explains why it takes so long to move from the “Eureka!” moment in the research lab to a “We can treat this” scenario at the hospital bed.

She knows first-hand, having worked with a University of Toronto team including Drs. Derek van der Kooy, Cindi Morshead, Brian Ballios and Michael Cooke that created a hydrogel to help transplanted stem cells thrive in the brain and eye. By injecting photoreceptor cells, the team was able to restore vision by approximately 15% in blind mice.

Their 2015 discovery garnered plenty of media attention, sparking hope that people with macular degeneration — a currently incurable condition — could have a new treatment. More than a year later, no such treatment is available or even on the horizon.

“For many people with macular degeneration, or any degenerative disease for that matter, studies like mine often produce a lot of hope, but also frustration that the ‘product’ is not available to them,” she writes in the Star.

As Dr. Shoichet explains, it can easily take 17 years or more to move a discovery from the lab to the patient. It takes lots of testing with animals before a new treatment can safely move to the three stages of human clinical trials, with all the attendant regulatory controls and approvals. It involves plenty of hard, painstaking work. And it costs lots of money.

But Dr. Shoichet remains optimistic. The opportunity to improve the lives of people with deadly diseases is what drives scientists to get up in the morning and get to work. As she writes, “I am optimistic we will deliver on the promise of regenerative medicine — but it won’t happen right away.”

“It’s a long road,” says the Dr. Wallace, Chair of the Vision Science Research Program at University Health Network’s Krembil Research Institute in Toronto. “Cell transplantation is tremendously complicated, difficult, and painstaking.”

But she is also optimistic about what the future holds for gaining a better understanding of the underlying causes of macular degeneration, which is triggered by deterioration of the cone photoreceptors (cones) in the centre of the eye that mediate reading and seeing fine detail. (In contrast, rod photoreceptors are concentrated along the edges of the retina to facilitate peripheral vision.)

“We are learning a lot about the biology that underpins photo cones, says Dr. Wallace. “They are a rare cell type and understanding their development has been difficult. Now we have tools to do that. That’s what I’m excited about.”

Her lab has developed a new mouse strain with fluorescently tagged cones to allow the researchers to track the progress of the cells to see if they can rescue visual function when transplanted into blind mice.

“We’re looking directly at this. Can cone cells transplant? Can they rescue vision? And if they can, what stage of cone development is the best for transplantation?”

The work she is doing feeds into a larger team effort dedicated to optimizing how to make photo receptors from human stem cells. What she learns from trying to engraft mouse cells will be applied to people. It also has implications for finding therapies for other neural degenerative diseases, such as Parkinson’s disease, Alzheimer’s and stroke.

“The eye has always been thought of as very different from the brain,” she says, “but it is part of the brain. Our work on identifying novel approaches to promote the survival of these cells could extend to promote neuron survival in other parts of the central nervous system.”

And the eye, aside from being the window to the soul, can also be a window into a person’s health. Says Dr. Wallace: “People are now using non-invasive imaging of the eye to look at markers of disease. There is a large study in Alzheimer’s that is imaging the eye to identify people at early stages of the disease. That’s happening more and more. People are appreciating that some aspects of eye health might inform the progression or even the diagnosis of other diseases.”

Dr. Wallace, who calls herself “a developmental biologist at heart,” doesn’t forget who she’s working for: people struggling with vision loss. Her past work with the Foundation Fighting Blindness, which helps fund her research, guarantees that. “I hear a lot about what’s important to patients.”

While progress is taking time, the science is steadily moving forward. “A lot of the things we’re doing now were not even being talked about 10 years ago.”

Dr. Duncan Stewart is one of the busiest people in regenerative medicine in Canada. An internationally respected cardiac specialist, he leads a three-city clinical trial investigating the potential benefits of genetically enhanced stem cells in healing severe heart attacks. …

Dr. Duncan Stewart is one of the busiest people in regenerative medicine in Canada. An internationally respected cardiac specialist, he leads a three-city clinical trial investigating the potential benefits of genetically enhanced stem cells in healing severe heart attacks. He also heads research at The Ottawa Hospital and teaches at the University of Ottawa. Dr. Stewart was recently named President and Scientific Director of the Ontario Institute for Regenerative Medicine (OIRM).

Q:Along with your research, clinical work and teaching, you’ve now taken on the role of Scientific Director for OIRM. How do you juggle it all?

A: It’s a matter of prioritization. My day job is here at the Ottawa Hospital Research Institute (OHRI) and that’s a fairly significant operation because we’re one of the largest in Canada now. I have a wonderful team here; it’s well set up and it can be done reasonably.

My clinical practice is really a hobby right now. I see patients in a clinic that specializes in what I’m interested in: pulmonary hypertension and serious cardiac heart problems. It’s personally rewarding; it keeps me sane and I can do something where I think I’ve made a difference. But it’s about a half-a-day a week. I don’t do much teaching … two or three or four courses a year.

I love doing research. I thought that when I took on these other responsibilities maybe I’d have to reduce that, but I’ve been successful at getting funding and the projects have been successful, so it’s larger. It’s aligned with the other things I do. The priorities of the OHRI are well aligned with the kind of research I do and the new opportunity with the OIRM, again, is very aligned.

Q:What are you excited about in the field of stem cells and regenerative medicine?

A: I’m excited about moving it more into the clinic. But we have to be realistic about the expectations. When we’re doing these clinical trials, we are doing them to learn. There are all kinds of issues we have to get right in order to unlock the real potential of these therapies. It’s going to take a fair bit of work and a number of clinical trials and going back to the bench to refine approaches before we get to the point where we can do the dramatic things we hope to do.

If you look at the pace of development for other disruptive technologies, in the early stages they didn’t look that fantastic. It’s similar to the early days of the personal computer. It was very frustrating. You did it for fun, really, for word processing, but it was hardly earth-shaking. Now you look at a PC and it can do what NASA used to do with a great big mainframe. A smartphone can do absolutely everything. The technology has evolved to the point where it’s changed the whole landscape.

I think that’s true also in the cell and gene therapy space. When I look at what’s going on in our lab and what’s going on globally, there is so much opportunity to advance this and improve the sophistication. The potential is just huge. What we’re doing in clinical trials, because we’re doing things that are well-tested and safe, tends to be using technologies that were developed 10 years ago. So it’s well behind the cutting edge of where the field is going. It takes that much time to get to clinical trials. I think what we’re looking for is more proof of principle, where we can — in a modest way — achieve some of our goals and learn where the opportunities are to make things better.

Q:So the key is to move into clinical trials?

A: It’s important even when you’re at the early stages of development of technologies, in a safe and responsible way, to get the experience in the context of the patient population that has the disease. Because you can never model all this completely in the lab. You may have a great technology, but unless you can use it in the real world patient population you could have a problem.

A good example is autologous cell therapy (therapies in which the patient’s own stem cells are used). For the chronic diseases we want to treat with regenerative approaches, we’re usually dealing with older patients who have risk factors and various diseases. All that reduces their regenerative activity of their own cells: their stem cells aren’t very good. So, you can do all the testing you want in young, healthy models but you’ve got to get at that problem. We need to find why these cells don’t work and what we can do to recover activity. Induced pluripotent stem cell technology (drawing cells from skin and reprogramming them back to an embryonic stem cell-like state) not only produces a pluripotent stem cell, it turns the age back to zero. There are other issues with that in terms of safety and whether it’s ready for prime time, but there are already clinical trials starting with that technology. That’s one way. There are other ways, maybe more subtle ways, to recover regenerative activity. These are the issues that come to the fore when you start to translate therapies into clinical trials.

Q:There is some frustration that things haven’t moved faster. Stem cells have been touted as being able to cure a number of diseases and that it’s going to happen soon. What’s holding the field back?

A: The frustration is probably our own doing: we bought into the hype or generated the hype too much. We need to set the expectations at a realistic level. It does no good to say we’re going to cure a disease with an approach when we’re not really there yet. It’s important that we do the clinical trials. It’s important to understand what benefit we get, if any, with a simple, early therapy so we can start improving it. But we need to understand that this is going to be a somewhat incremental process at the beginning. It’s not going to be earth-shattering and necessarily curative in the first iteration. Setting that expectation will decrease that frustration.

The reality is, this takes time. It’s a very different activity to be moving these approaches into clinical trials than doing the fundamental research. People who have made major discoveries are not necessarily equipped or have the ability or interest to move them into clinical trials.

What does work well is a team approach. You have the innovative scientist who makes the discovery as an important part of that team. But a lot of what has to be done to translate that into a clinical trial is the preclinical research needed for regulatory submissions — which is not the exciting kind you publish in Nature. It’s a different skill set than is normal in a discovery lab. And you need people who understand the disease and where the therapy should best be targeted. You need people who understand what kind of trial needs to be done to get the right answers. It takes a lot of people who have complementary expertise who come together in a translational research program and say ‘OK, we think this is potentially going to make a big difference. This is how we’re going to move it forward in a safe, responsible and effective manner.’ Coincidentally, this is exactly what OIRM is doing, funding these team grants to develop translational teams.

Q:What do you see as OIRM’s role?

A: Ontario is a big player in Canada in this space. We’re not the whole story but were a big part of the story. There is tremendous talent. What OIRM can do is help people understand how to move this forward, how to build these teams. It’s education: bringing in experts who have had success and helping these teams move forward, helping them build in the areas where they need to build. It’s not always intuitive. I see OIRM as a real catalyst. It also has a role to shepherd these activities in the right direction so there’s the greatest chance of success.

International stem cell scientists, led in Canada by Dr. John Dick and in the Netherlands by Dr. Gerald de Haan, have found the switch to harness the power of cord blood and potentially increase the supply of stem cells for cancer patients needing transplants.…

International stem cell scientists, led in Canada by Dr. John Dick and in the Netherlands by Dr. Gerald de Haan, have found the switch to harness the power of cord blood and potentially increase the supply of stem cells for cancer patients needing transplants.

The findings, published today in Cell Stem Cell, could provide a way to make more stem cells from cord blood, which is increasingly available through public cord blood banking.

“Stem cells are rare in cord blood and often there are not enough present in a typical collection to be useful for human transplantation,” says Dr. Dick, Senior Scientist, Princess Margaret Cancer Centre, University Health Network (UHN), in a media release.

“Our discovery shows a method that could be harnessed over the long term into a clinical therapy and we could take advantage of cord blood being collected in various public banks that are now growing across the country.”

The Dick-de Haan teams found that when a stem cell divides it makes progenitor cells that retain key properties of being able to develop into any one of the 10 mature blood cell types, but they have lost the stem cell’s key ability to self-renew.

Working with mice and human models of blood development, the teams identified microRNA (mirR-125a), a genetic switch that is normally on in stem cells and controls self-renewal, gets switched off in the progenitor cells.

“Our work shows that if we artificially throw the switch on in those downstream cells, we can endow them with stemness and they basically become stem cells and can be maintained over the long term,” says Dr. Dick.

This is just the latest discovery by Dr. Dick. He isolated a human blood stem cell in its purest form and was the first to identify cancer stem cells in leukemia and colon cancer.

Dr. de Haan is Scientific Co-Director, European Institute for the Biology of Ageing, University Medical Centre Groningen, the Netherlands.

Is it possible that stem cells will some day repair brain tissue damaged by tumours, epilepsy or injuries?

The Ontario Institute of Regenerative Medicine and the Ontario Brain Institute are hosting a free public event Tuesday, July 12 to shed light on possibilities, provide information on stem cell research and discuss realistically what the future holds. …

Is it possible that stem cells will some day repair brain tissue damaged by tumours, epilepsy or injuries?

The Ontario Institute of Regenerative Medicine and the Ontario Brain Institute are hosting a free public event Tuesday, July 12 to shed light on possibilities, provide information on stem cell research and discuss realistically what the future holds. It begins at 6:30 p.m. at the Toronto Reference Library and features neuroscientist/researcher Dr. Cindi Morshead of the University of Toronto and neurosurgeon/researcher Dr. Michael Fehlings of University Health Network.

This is an area where Canadian researchers excel: neural stem cells were discovered in Canada by Dr. Sam Weiss at the University of Calgary. Research into the use of stem cells to regenerate damaged brain cells is in the early stages, but has many potential applications for neurological diseases and damage including stroke and spinal cord repair.

Admission is free but space is limited. For more information, and to register click here.

Japan believes regenerative medicine will grow from a $950-million domestic industry in 2020 to a $10 billion one by 2030, according to a report by Bloomberg News.…

Japan believes regenerative medicine will grow from a $950-million domestic industry in 2020 to a $10 billion one by 2030, according to a report by Bloomberg News.

And the Japanese expect to tap into a $120-billion global market over the same time span if regenerative medicine fulfills its potential to set off “a medical and industrial revolution.”

The Bloomberg report illustrates how Japan is building on Nobel Prize winner Dr. Shinya Yamanaka’s discovery of induced pluripotent stem cells (iPS cells) to help recharge its economy. The government has allocated $1 billion in funding and streamlined regulations to expedite the movement of research to the clinic. Meanwhile industrial heavyweights like Fujifilm and Hitachi Ltd. are moving away from fading product lines and diminishing markets to invest in new technology and products using Dr. Yamanaka’s discovery.

“Japan has taken a bold step,” Dr. Hardy TS Kagimoto, who heads the Healios KK biotech firm, told Bloomberg. “It’s been a while since our country has had innovative companies in a global industry that can help us maintain economic power, and we think regenerative medicine can be the one.’’

The powerful iPS cells are made by reprogramming adult skin cells back to an embryonic-like state, a process that circumvents ethical concerns over the use of embryonic stem cells. Beyond transplant purposes, the cells can be used to screen drugs, which Dr Yamanaka believes could “facilitate drug development tremendously.”

Drug regulators in Japan, Europe and U.S. are expected to release coordinated draft drug guidelines for the use of iPS cells in pre-clinical trials by the end of 2017, according to the article, which “could upend the entire market.”

Canada, where stem cells were discovered in the 1960s, is a leader in stem cell research and development but is at risk of losing ground. James Price, President and CEO of the Canadian Stem Cell Foundation (CSCF) recently authored an iPolitics article calling for a comprehensive national approach:

“In order to maintain our position as a global leader in the field that we discovered and pioneered, to help thousands of Canadians and their loved ones who are struggling with life-threatening conditions, and to transform the stem cell sector into a thriving industry built on of high-quality jobs that support families across the country, we need a truly national stem cell effort.”

The CSCF advocates for the Canadian Stem Cell Strategy, an innovative private-public partnership that is the product of consultations with 150 scientists, medical professionals, leaders from major health charities, industry experts and philanthropists. The goal of the Strategy is to deliver up to 10 new therapies in 10 years while helping to grow the Canadian economy and create 12,000 new jobs.

Jim Gass, a 66-year-old lawyer who now lives in San Diego, has inadvertently become the human face of the dangers of stem cell tourism.…

Jim Gass, a 66-year-old lawyer who now lives in San Diego, has inadvertently become the human face of the dangers of stem cell tourism.

A feature in the New York Times headlined “A Cautionary Tale of Stem Cell Therapy Abroad” tells of how Mr. Gass spent $300,000 by travelling to clinics in Mexico, China, and Argentina in search of a miracle stem cell cure after he had a stroke. Recently, his American doctors found a huge mass of someone else’s cells growing aggressively in his lower spine.

As the article explains, Mr Gass travelled from clinic to clinic believing that the worst that could happen was there would be no improvement in his post-stroke condition, which left his left arm useless and his left leg weak. Except for being able to use his right arm, he is now paralyzed from the neck down.

In Mexico he received an injection of fetal cells shipped from Russia that, for a time, seemed to improve his ability to walk: “Then something disturbing happened,” Mr. Gass told the Times. “I felt pain when I would lie down, like I was lying on a tumor. “I started to lose my ability to walk and I fell down a lot.”

That’s when Boston doctors found the mass containing someone else’s primitive cells. They treated it with radiation, which seemed to slow its growth, but the mass is growing again.

As discussed on the Treatments Abroad page on our website, scientific discoveries and innovations surrounding the potential of stem cell science have led to great enthusiasm about potential benefits to patients. But the dark side of that enthusiasm is the hype, exaggerated publicity and inaccurate claims in the interest of financial gain.

Many unregulated clinics around the world offer treatments that are simply not based on sound scientific evidence. If you or someone you know is considering such a therapy, you’d be wise to consult a booklet produced by the University of Alberta Faculty of Law, Albany Medical College and the Stem Cell Network entitled What you need to know about stem cell therapies. A PDF version is available here.

An excellent web resource is the International Society of Stem Cell Research Web page: A Closer look at Stem Cells. It includes a Patient Handbook downloadable in several languages.

Both sources advise potential patients to be wary of clinics that offer patient testimonials in place of evidence gathered through rigorous clinical trials. Or, as Mr. Gass now says: “Don’t trust anecdotes.”

Dr. Allen Eaves, a member of the Canadian Stem Cell Foundation’s Board of Directors and a founding Till & McCulloch Leadership Circle member, is being appointed to the Order of British Columbia — the province’s highest form of recognition.…

Dr. Allen Eaves, a member of the Canadian Stem Cell Foundation’s Board of Directors and a founding Till & McCulloch Leadership Circle member, is being appointed to the Order of British Columbia — the province’s highest form of recognition.

An internationally respected leukemia researcher and clinician, Dr. Eaves is the founder and owner of Vancouver’s STEMCELL Technologies Inc., Canada’s largest biotech company. He co-founded the Terry Fox Laboratory and served as its director for 25 years as well as heading the BC Cancer Agency for 18 years, developing one of the first bone marrow transplant programs in the world.

Called the “cell-made man” by BC media, Dr. Eaves began STEMCELL in 1993 with a staff of eight. He now employs more than 800 people. STEMCELL develops specialty cell culture media, cell separation products and ancillary reagents for life science research and delivers them to scientists around the world. In 2015 STEMCELL was named Life Sciences Company of the Year by LifeSciences BC.

Dr. Eaves has a strong incentive to continue his efforts, telling BCBusiness in 2013 that “I want t to cure cancer. That’s the motivation,” he said. “It’s all about curing some of these diseases, wiping them off the face of the earth.”

The investiture will take place July 26 in Victoria. To read more about Dr. Eaves, click here.

Want to know more about last Thursday’s announcement that two Ottawa doctors have found a way to shut down aggressive MS?…

Want to know more about last Thursday’s announcement that two Ottawa doctors have found a way to shut down aggressive MS?

In case you missed it, Rita Celli, host of CBC’s Ontario Today, dedicated her entire hour-long program on Friday to discussing the report. Published in the Lancet, the paper details how Drs. Harry Atkins and Mark Freedman were able to halt the progression of the disease for the 24 patients in the study. Some patients, like Jennifer Molson, saw their MS symptoms disappear entirely over time. You can access the podcast here.

Ms. Celli features Dr. Freedman who explains how the treatment destroys the patient’s immune system through chemo and then rebuilds a new MS free one using their own previously harvested bone marrow stem cells. He also takes calls from listeners, some of whom tell their stories of life with MS.

The program also features clips of Ms. Molson explaining how the treatment freed her from life in a wheelchair, and Dr. Atkins declaring that “MS can be stopped in its tracks.”

The program provided a comprehensive look at the treatment, which is considered high risk (one patient died) and is only for those MS patients for whom nothing else is working.

A team led by two doctors from The Ottawa Hospital has proved that stem cells, used in combination with chemotherapy can halt the progression of aggressive multiple sclerosis (MS) and — in some cases — help patients recover from the autoimmune disease.…

A team led by two doctors from The Ottawa Hospital has proved that stem cells, used in combination with chemotherapy can halt the progression of aggressive multiple sclerosis (MS) and — in some cases — help patients recover from the autoimmune disease.

In a paper published today in the prestigious medical journal The Lancet, Drs. Harry Atkins and Mark Freedman show that bone marrow stem cell transplants can eliminate all signs of damaging brain inflammation by rebuilding a patient’s immune system.

MS occurs when a person’s own immune system attacks the myelin protective sheath that covers nerves, causing inflammation and damage that inhibits communication between cells in the nervous system — the brain and spinal column.

The researchers took bone marrow stem cells from patients with aggressive MS and purified them in a laboratory. After the patients underwent extreme chemotherapy, their robust stem cells were returned to rebuild new immune systems.

The clinical trial involved 24 patients and tracked their progression over several years. According to The Ottawa Hospital’s news release, after the treatment:

Not a single participant experienced a clinical relapse (zero relapses in 179 patient-years), whereas before treatment, the participants experienced an average of 1.2 relapses per year.

Not a single new active inflammatory lesion could be detected in the brains of any of the participants.

Not a single participant required MS-specific drugs to control their disease.

70% of participants experienced a complete stop in disease progression.

The average rate of brain shrinkage, typically a measure that correlates with MS progression, returned to levels associated with normal aging.

40% of participants experienced some lasting reversal of such symptoms as vision loss, muscle weakness and balance problems.

Some participants were able to return to work or school, regain the ability to drive, get married and have children.

In Jennifer Molson’s case, the treatment eradicated all traces of the MS that had taken over her life. Prior to taking part in the study, she was receiving 24-hour care at the Rehab Centre at the Ottawa Hospital, “learning to how to live with my disability.” She had quit her job and could only walk with the help of forearm crutches or a walker. Life in a wheelchair was imminent.

Now free of MS symptoms for more than a dozen years, Ms. Molson has resumed a demanding career and a busy schedule. As she described in the book Dreams & Due Diligence: Till and McCulloch’s Stem Cell Discovery and Legacy, “I downhill ski, I drive a standard. I can skate. I can dance … Am I cured? I like to use that word. They (Drs. Atkins and Freedman) don’t like to use that word. They’re calling it a lasting remission. I’m very lucky. I got a second chance at life.”

While his recovery was less dramatic, Vancouver’s Aaron Prentice said his quality of life is much improved: “I am now five years post-transplant,” he wrote in the Foundation’s Cellections newsletter last year. “I have not had a relapse and no longer require a cane. My gait has improved significantly and continues to do so. My arms are symptom free.”

Marjorie Bowman, trial coordinator and advanced practice nurse at The Ottawa Hospital, expressed her admiration for the remarkable “courage and dedication” demonstrated by the trial’s participants. “We thank the patients from across Canada who participated in this clinical trial, as well as their family members,” she said.

The treatment, Dr. Atkins explained in Dreams & Due Diligence, is a variation on the bone marrow ablation that leukemia patients undergo.

“MS is an autoimmune disease where the immune system is attacking a patient’s brain. The simple concept behind our treatment is, ‘Let’s just get rid of the old immune system and put back the seeds, let a new one grow and hope that it won’t learn the same lesson.’

“Because stem cells don’t carry over immunologic memory. That’s really what we have tried to do. We had a track record for doing transplants for leukemia and knew how we could damage the immune system to remove it. We just applied the lessons we learned in care of patients with leukemia and applied them to this new setting.”

Not for everyone

However, the therapy is not for all MS patients.

“It is only used in very severe cases because participants face a significant risk of infection and other side-effects, including death,” Dr. Atkins said in the media release. “The risks are similar to those faced by leukemia patients undergoing this kind of treatment.”

Indeed, one participant in this study died of liver failure due to the treatment and another required intensive care for liver complications. The treatment regimen was modified to reduce toxicity, but all participants still developed fevers, which were frequently associated with infections.

People who are interested in this therapy should speak with their own neurologist, who can request a referral to The Ottawa Hospital MS Clinic or another major hospital with experience in this area. The Ottawa Hospital cannot treat people without valid Canadian health coverage.

And Dr. Freedman has gone on to co-lead a new chemo-free MS clinical trial using mesenchymal stem cells. “These cells have been shown, at least in early studies in humans, to repair — period,” he explained recently. “But they happen, at the same time, to have an anti-inflammatory effect. So they may be able to accomplish both things together. Without the need of any chemo, there is very little risk to the people taking it.”

A turning point for MS

Yves Savoie, President and CEO, MS Society of Canada, called publication of the paper “a turning point” in MS care. “What started as a bold idea has translated into a treatment option for people living with highly active, relapsing MS.”

The $6.47 million trial was funded by the MS Society of Canada and its affiliated Multiple Sclerosis Scientific Research Foundation. The research was also supported by The Ottawa Hospital Foundation, The Ottawa Hospital Department of Medicine and Canadian Blood Services.

The Ontario Institute for Regenerative Medicine (OIRM) today named Dr. Duncan Stewart, one of Canada’s leading stem cell researchers, as its new President and Scientific Director.…

The Ontario Institute for Regenerative Medicine (OIRM) today named Dr. Duncan Stewart, one of Canada’s leading stem cell researchers, as its new President and Scientific Director.

“It will be my pleasure to serve OIRM by helping the organization, its researchers, trainees and staff to fulfill their passion to make a difference for all Ontarians,” Dr. Stewart said in a media release.

He succeeds Dr. Janet Rossant, who launched OIRM in 2014 but recently took on a new role as the Gairdner Foundation’s President and Scientific Director. She praised Dr. Stewart “the perfect choice to lead OIRM as it moves into the next phase of growth.”

Dr. Stewart will remain in Ottawa to pursue his lab and clinical research activities and to carry on as Executive Vice-President of Research at The Ottawa Hospital.

“Given the breadth of his skills, Duncan brings unique perspectives on the regenerative medicine environment, particularly in the critical area of clinical trials and the development of new treatments, which is a key part of our mission,” said Sharon Colle, Chair of the OIRM Board and President and CEO, of The Foundation Fighting Blindness.

Congratulations to Dr. Janet Rossant on her investiture as a Companion in the Order of Canada (CC) in a ceremony presided over by Governor General David Johnston.…

Congratulations to Dr. Janet Rossant on her investiture as a Companion in the Order of Canada (CC) in a ceremony presided over by Governor General David Johnston.

Dr. Rossant, who chairs the Canadian Stem Cell Foundation’s Science Leadership Council, was one of only two Canadians to be honoured as a Companion along with Nobel Prize winning scientist Dr.Arthur McDonald. The three tiers of the order are: Companion, Officer, and Member. Friday’s ceremony also included four Officers and 42 Members.

A Rideau Hall news release noted that Dr. Rossant established entirely new concepts in developmental biology: “As a professor at the University of Toronto, she has characterized genes that are critical to the earliest stages of embryonic development and discovered control systems that enable cell differentiation. Her globally renowned research is foundational to the development of new treatments for a range of conditions including cancer and degenerative diseases.”

Dr. Rossant is a Senior Scientist & Chief of Research Emeritus at SickKids Research Institute. She was the inaugural President & Director of the Ontario Institute of Regenerative Medicine and recently became the Gairdner Foundation’s President and Scientific Director.

Across Ontario, hundreds of people have a stronger appreciation of stem cells, thanks to the Ontario Institute for Regenerative Medicine’s (OIRM) Stem Cell 101 — The Promise and Potential.

The spring lecture series — featuring world-leading scientists explaining the field, talking about their research and discussing the ethical issues involved — concluded last night in Ottawa where an audience of about 100 listened, asked thoughtful questions and exchanged ideas with the experts.

Co-sponsored by the University of Ottawa and The Ottawa Hospital, Tuesday’s event followed similar free-to-the-public sessions in London, Hamilton and Toronto held in partnership with Western University, McMaster University and SickKids Hospital, respectively.

At each event, local scientists addressed such questions as: “What is a stem cell?” “What’s new in stem cell research?” “What kinds of treatments are using stem cells today?” “What is stem cell tourism?”

At last night’s session, Dr. Bill Stanford, a senior scientist at The Ottawa Hospital, told the audience how he left an uncertain regulatory environment in the United States, where President George W. Bush had imposed a ban on federal funding for research on new embryonic stem cell lines, to come to work in Canada. President Barack Obama lifted the ban, but Dr. Stanford chose to stay in Ottawa, he told CBC News, because “here in Ottawa people work together instead of working by themselves and that makes things go much faster and better.”

As part of his discussion on ethics, Dr Jeff Blackmer, Vice-President of Medical Professionalism at the Canadian Medical Association (CMA), focused on private versus public banking of umbilical cord blood, which provides a rich and non-controversial supply of stem cells for transplant. He noted that private cord blood banks, which charge significant collection fees and annual storage fees, can contain an implied messages in their advertising that parents who don’t use their services are letting their children down. While such a decision is a personal one, he noted that public cord blood banking reflects the spirit of universal health care. Canada’s Cord Blood Bank now operates in four cities.

Asked about when new therapies will make it to the clinic, The Ottawa Hospital’s Dr. Harry Atkins said that while the field is progressing rapidly, significant technological challenges remain. That makes it difficult to predict whether a new treatment might become available, he said, noting that in some cases “it could be two years, it could be a decade.”

(Note: The Spring edition of our Cellections newsletter features some of the leading-edge stem cell research underway in Ottawa. Subscribe here, by clicking on “Newsletter.”)

One thousand Canadians need a stem cell donation on any given day, according to Canadian Blood Services (CBS). …

Q&A with Dr. Heidi Elmoazzen

One thousand Canadians need a stem cell donation on any given day, according to Canadian Blood Services (CBS). About 250 usually can find a match in their own family. The other 750 must look elsewhere. For years, CBS has operated its OneMatch Stem Cell and Marrow Network to find and match volunteer donors to patients. Last year, CBS added to the much needed supply of donor stems cells by officially launching its Cord Blood Bank where new mothers can donate umbilical cord blood — a plentiful source of stem cells that are more easily matched than adult stem cells. The bank is supported by nine provinces and the three territories (Québec has its own cord blood bank and registry). Five collection sites now operate in four cities. Dr. Heidi Elmoazzen is the Director of the Cord Blood Bank.

Q: The official launch was last year, but how long has the national public cord blood bank been operating?

A: We started collecting in Ottawa in 2013, then rolled out to Brampton in 2014. We started collections in Edmonton and Vancouver in 2015. We did the official launch in June of 2015 to say, “We’re open.”

Q: Canada was the last G7 country to establish a public cord blood bank. Why were we so late to the party?

A: Part of it is just the way funding is structured. We needed agreement from the provinces and territories. But there are actually advantages of us coming late to the party. We’ve been able to learn from other cord blood banks and we’re fortunate to now be in a position where we’ve set up one of the best in the world in terms of quality of inventory.

Q: What stage are you at now?

A: Our biggest goal is building up an inventory that’s reflective of the unique population we have here in Canada. We have a lot of ethnic groups that you don’t find in other parts of the world, including our Aboriginal — First Nations, Métis and Inuit — populations. We have a lot of mixed race patients here. It’s building up that inventory that reflects that unique population. And we’re really going for quality units — large units with lots of stem cells in them so there are enough stem cells for a transplant. As you can appreciate, we need an inventory of a certain size before we see uptake. We’re in that phase now. We currently have 1,145 units listed and available to Canadian and international transplant centres. We’ve had inquiries, but nothing has been shipped out yet. So that’s our next big milestone. I’m expecting we will probably see that soon.

Q: So, for example, a Canadian patient with a blood-based cancer who is looking for a stem cell transplant, they would look at what your bank has available and look internationally as well?

A: It goes through our OneMatch Stem Cell and Marrow Network, where we search all the adult donors and cord blood units in Canada as well as internationally. We have access to over 27 million adult donors around the world. In Canada there are 345,000 adult donors. And then you have 684,000 publicly banked (cord blood) units, with our own units in there as well.

Q: Are most people aware that the public bank is there and there’s an option to donate cord blood?

A: There are always opportunities to increase awareness. We really try to make moms who are delivering at one of our designated collection hospitals aware. We have conversations with all of the physicians who have delivering rights at those hospitals. We try to do outreach so that moms do know this is available to them.

Q: What’s their reaction been?

A: It has been great. It’s a very easy sell to mothers because they know the alternative is the cord blood will be discarded as medical waste. We don’t get push-back.

Q: The collection service is only available in four centres. What happens if you don’t happen to live in Ottawa, Brampton, Edmonton and Vancouver?

A: Unfortunately, then you can’t participate in the public bank. But it’s important to realize these units are available to anyone in Canada and around the world who needs them. We selectively chose the cities we are in and what hospitals we partner with. We wanted large urban centres with a large ethnic diversity. We wanted hospitals that had at least 4,000 births (per year) with a minimum 20% ethnic diversity. We’re trying to build a bank that reflects the ethnic diversity in Canada.

Q: Will there be more collection centres opening?

A: We’re always looking at whether we need to do that. At this time there are no plans to open any more sites.

Q: The total price tag for the bank is $48 million, with $12.5 million raised in a fundraising campaign and the rest coming from the provinces and territories. Why did one-quarter of the funds come from fundraising?

A: It was to show the governments that we had skin in the game and that Canadian Blood Services was committed to this.

Q: Does this save money? If a Canadian gets a stem cell transplant from here in Canada, does that save money compared to going to Europe to have the stem cells harvested and shipped over here?

A: Absolutely. It costs about $40,000 when we use either an adult donor or a cord blood unit from overseas. It’s a huge cost savings to the health care system, especially when you’re talking about adults because they’re using two cord blood units per transplant. The fact that these are available through our transplant centres at no cost represents a huge savings.

Q: Can the cord blood also be used for research?

A: Yes; we have a cord blood for research program. Moms who donate in Ottawa can indicate on their permission to collect form that in the event that their baby’s cord blood is not bankable it can go to research. We make those research units available to scientists across Canada. We have shipped out hundreds of units for research purposes. There’s been a big uptake because it’s such a valuable resource.

When babies are born prematurely — especially at fewer than 28 weeks in the womb — their lungs are not fully formed and they need help to breathe. …

Dr. Bernard Thébaud

When babies are born prematurely — especially at fewer than 28 weeks in the womb — their lungs are not fully formed and they need help to breathe. At neonatal units in hospitals around the world, doctors put these babies on ventilators to give them additional oxygen.

Unfortunately, the very device that keeps these tiny babies alive can create life-long consequences by triggering bronchopulmonary dysplasia (BPD).

“Approximately 40% of extreme pre-term babies will get BPD,” says Dr. Bernard Thébaud, a researcher and clinician with The Ottawa Hospital and the Children’s Hospital of Eastern Ontario. “They will have a higher risk of developing asthma and of being re-hospitalized after being sent home. They may be at risk of early onset aging of their lungs — something similar to smoker’s lung.”

There is no treatment for BPD. Steroids that decrease the inflammation it causes can also inhibit brain development. “So we’re very cautious in administering steroids,” says Dr. Thébaud, who is working on a stem cell solution to the ventilator/BPD conundrum.

In mesenchymal stem cells extracted from Wharton’s jelly, a gelatinous substance within the umbilical cord, Dr. Thébaud hopes to find a BPD treatment that will also help other organs thrive.

“These cells will do several things. They will attenuate the inflammation, but also promote lung growth. In addition, we believe their benefits will not be limited to the lung. They will also have some beneficial effects on the brain, the gut and other organs in preterm babies that still need to grow and need to be protected from injury. “

So far, Dr. Thébaud, who is also a professor of pediatrics at the University of Ottawa where his holds the uOttawa Partnership Research Chair in Regenerative Medicine, has only tested the mesenchymal stem cells on animals. But the results have been encouraging, with the stem cells protecting the lungs from oxygen induced injuries. “We still have to do further tests with the cells made in a Good Manufacturing Process facility for a clinical grade product.”

He looks forward to conducting a Phase 1 clinical trial in the near future to test the feasibility and safety in newborns. “If it’s safe, we’ll move to Phase 2 and enroll more babies.”

As a researcher who cares for these fragile babies every day, Dr. Thébaud is both frustrated and hopeful.

“When we see these babies in our unit and we discuss what we can offer, we can see how the disease evolves and we know that we will be stuck at one point. Sometimes a nurse will come up to me and say, ‘Oh, Bernard, this baby needs your stem cells. Can you bring some over from our lab?’ On one hand it’s frustrating. On the other, it’s very rewarding to see that everyone recognizes the potential of this therapy and is waiting for it.”

The beauty of these mesenchymal stem cells, also called stromal cells, is that they are easily harvested from umbilical cords that would otherwise be discarded as medical waste. They are also easy to expand in a lab, generating the millions of cells required for transplant. Because no genetic match is required between donor and recipient, they can be supplied off-the-shelf. And they are powerful.

“Because the cells are young, they might be more potent than bone-marrow-derived mesenchymal stem cells,” says Dr. Thébaud. “So it could be that the product we are developing for the babies could also be beneficial in treating adult diseases.” and superior to current bone marrow-derived stem cells.”

Sometimes a picture is worth so much more than words. That’s the case with trying to explain the current crop of clinical trials for stem cell therapies.…

Sometimes a picture is worth so much more than words. That’s the case with trying to explain the current crop of clinical trials for stem cell therapies. Our partners at the Ontario Institute for Regenerative Medicine — who are sponsoring the Stem Cells 101 event in Ottawa on Tuesday May 10 — have produced a helpful graphic that highlights the nine clinical trials now underway across Ontario. Our challenge will be to produce a national version. But in the meantime, here’s what’s happening in Ontario:

The fourth and final event in the Ontario Institute for Regenerative Medicine’s series Stem Cells 101: The Promise and Potential is set for Tuesday, May 10 at the Civic Campus of the Ottawa General Hospital.…

Want to know more about stem cells and regenerative medicine?

The fourth and final event in the Ontario Institute for Regenerative Medicine’s series Stem Cells 101: The Promise and Potential is set for Tuesday, May 10 at the Civic Campus of the Ottawa General Hospital.

Like the three previous events in London, Hamilton and Toronto, the Ottawa forum features leading scientists talking about their work and discussing everything from ‘What is a stem cell?’ to ‘What is stem cell tourism?’.

The series has been well attended with full or near-full houses at most of the venues. Organizers of The Ottawa Hospital session (details here) also expect a big crowd, though seats are still available.

Small wonder there is big interest: Dr. Harry Atkins, who is doing world-leading work in using stem cells to fight multiple sclerosis, is one of the three featured speakers. Dr. Bill Stanford, Senior Scientist at the Sprott Centre for Stem Cell Research, is offering a crash course on stem cell biology and Dr. Jeff Blackmer, Vice-President of Medical Professionalism at the Canadian Medical Association, will tackle the ethical issues in stem cell research and application.

The Ottawa event begins a 7 p.m. and runs about 90 minutes. Free parking is available.

Researchers believe they have found a key piece to the puzzle of brain tumour formation.

A study published today in Nature Neuroscience shows that glioblastoma tumours need a protein called OSMR (Oncostatin M Receptor) to form.…

Researchers believe they have found a key piece to the puzzle of brain tumour formation.

A study published today in Nature Neuroscience shows that glioblastoma tumours need a protein called OSMR (Oncostatin M Receptor) to form. Glioblastoma is one of the most deadly cancers, resistant to radiation, chemotherapy and difficult to remove with surgery.

“The fact that most patients with these brain tumours live only 16 months is just heartbreaking,” said Dr. Arezu Jahani-Asl, lead author of the study. Dr. Jahani-Asl, an assistant professor at McGill University and a principal investigator at the Jewish General Hospital, did much of the research while she was a postdoctoral fellow co supervised by Dr. Michael Rudnicki at The Ottawa Hospital and the University of Ottawa and by Dr. Azad Bonni from Harvard Medical School and Washington University School of Medicine.

“Right now there is no effective treatment, and that’s what drives me to study this disease,” said Dr. Jahani-Asl.

The research team studied human brain tumour stem cells taken from glioblastoma patients. While it was previously believed that any cancer cell could reproduce to form a whole tumour, researchers have since learned that in brain cancer only a few kinds of cells have this ability. If a single one of these brain tumour stem cells is left behind after surgery, it can create a whole new tumour. Working with mice, the research team found that blocking OSMR activity in these cells prevented them from forming brain tumours.

“Being able to stop tumour formation entirely was a dramatic and stunning result,” said Dr. Rudnicki, senior co corresponding author of the study. “It means that this protein is a key piece of the puzzle, and could be a possible target for future treatments.”

Dr. Bonni, senior co-corresponding author, said that while the results are exciting, there is much more work to be done. “The next step is to find small molecules or antibodies that can shut down the protein OSMR or stop it from interacting with EGFR (the epidermal growth factor receptor that drives tumour formation in glioblastoma). But any human treatment targeting this protein is years away.”

If everything you’ve ever heard was true, cure-all stem cells would soon be available at your corner drug store or clinic.…

If everything you’ve ever heard was true, cure-all stem cells would soon be available at your corner drug store or clinic.

But it’s not that simple.

Which is why the Ontario Institute for Regenerative Medicine is co-sponsoring Stem Cells 101 — a series of free public events in four Ontario cities beginning next week.

The sessions, held in partnership with Western University, McMaster University, SickKids Research Institute, the University of Ottawa and The Ottawa Hospital, are geared to provide the general public with hype-free, up-to-date, science-based information on stem cells and regenerative medicine: Such as:

What are these incredible cells?

What stem cell therapies are currently available and what’s on the horizon?

How are we getting to the next big breakthrough and what are the societal considerations for this research?

The sessions feature top Canadian researchers and clinicians explaining their work and talking about the potential and possibilities. Here’s the lineup:

Introduction to the ethical, legal and social issues surrounding stem cells. Dr. Ubaka Ogbogu, assistant professor in the Faculties of Law and Pharmacy & Pharmaceutical Sciences at the University of Alberta.

A 73-year-old man from Hawkesbury, Ontario, survived a deadly infection after receiving millions of mesenchymal stem cells in a world-first trial at The Ottawa Hospital.…

A 73-year-old man from Hawkesbury, Ontario, survived a deadly infection after receiving millions of mesenchymal stem cells in a world-first trial at The Ottawa Hospital.

Charles Berniqué developed severe septic shock in June of last year after his esophagus burst, likely from food poisoning. Septic shock is a deadly condition in which rampant infection triggers hyper-activation of the immune system, causing the cardiovascular system and organs to fail.

Mr. Berniqué was first treated by thoracic surgeons, who restored his fluids, repaired his esophagus and started antibiotic therapy. He was placed into a coma in the intensive care unit where mechanical ventilation and dialysis supported his heart, lungs and kidneys.

During this time, his wife Maureen consented to his participation in the clinical trial led by Drs. Duncan Stewart and Lauralyn McIntyre.

Within 24 hours, Mr. Berniqué received an intravenous infusion of 30 million mesenchymal stem cells originally extracted from the bone marrow of a healthy Ottawa volunteer.

“Our laboratory studies that showed that mesenchymal stem cell therapy tripled survival in a mouse model of septic shock,” Dr. Stewart, Executive Vice-President of Research and senior scientist at The Ottawa Hospital, said in a media release. “The cells also reduced damaging inflammation and helped the mice eliminate the bacteria.”

Dr. McIntyre, an intensive care physician and senior scientist at The Ottawa Hospital, was impressed by the results. “Researchers around the world have spent decades trying to find a therapy that will treat the root causes of septic shock rather than just the symptoms, but so far, none of these therapies have improved survival,” she said. “We don’t know whether the cell therapy played any role in Mr. Berniqué’ s remarkable recovery, but the cells were very well tolerated and we are excited to continue to study this promising therapy in more patients.”

Mesenchymal stem cells have been studied extensively in human clinical trials for other conditions, but The Ottawa Hospital trial is the first in the world to evaluate the cells specifically for the treatment of septic shock. The main goal of the Phase I trial is to evaluate the tolerability and feasibility of the cells. However, the researchers have already received funding from the Ontario Institute for Regenerative Medicine to begin scaling up their cell bank for a larger Phase II trial, which will help determine if the therapy is effective against septic shock.

As for Mr. Berniqué, he’s grateful to be alive and happy that the care he received may do the same for others. “It is tremendous what The Ottawa Hospital did for me,” he said. “I was so close to death, but I received the best care in the world and got to participate in this study which could help many people.”

Tuesday’s federal budget bodes well for stem cell research and development in Canada.

First, there is the Government of Canada’s commitment to make available up to $800 million to support innovation networks and clusters as part of the Government’s upcoming Innovation Agenda.…

Tuesday’s federal budget bodes well for stem cell research and development in Canada.

First, there is the Government of Canada’s commitment to make available up to $800 million to support innovation networks and clusters as part of the Government’s upcoming Innovation Agenda.

Finance Minister Bill Morneau and the Government indicated they recognize the need to “catalyze private sector dynamism, generate greater value from public investments in innovation and enable the pursuit of ambitious initiatives that bring a critical mass of stakeholders together and connect their ideas to the marketplace.”

This approach — catalyzing private sector dynamism — reflects the essence of the proposed Canadian Stem Cell Strategy, the privately led plan that unites all the players in Canada’s stem cell sector to deliver up to 10 new curative therapies within 10 years. Crafted in consultation with 150 scientists, doctors, leaders from health charities, industry experts and philanthropists, the Strategy calls for a one-third investment by the Government to be matched by two-thirds private and non-federal funds.

Second, Budget 2016 allocates $12 million over two years to support the Stem Cell Network in its research, training and outreach activities. The Network, whose funding was scheduled to end next year, aligns the efforts of stem cell scientists across Canada. Providing funding for the Network further strengthens the case for a comprehensive national strategy for stem cell R&D.

“Canada’s stem cell community appreciates the recognition in Budget 2016 and looks forward to working with the Government as it builds its Innovation Agenda,” says James Price, President & CEO of the Canadian Stem Cell Foundation. “The proposed Strategy will improve the health of Canadians, leverage our unique history and incredible experience in this sector and secure Canadian leadership in the next wave of modern medicine.”

A single injection of stem cells can reverse age-related osteoporosis in mice, a team of Canadian researchers has shown.

The discovery provides hope of some day developing a treatment for the crippling disease that affects 200 million people worldwide.…

A single injection of stem cells can reverse age-related osteoporosis in mice, a team of Canadian researchers has shown.

The discovery provides hope of some day developing a treatment for the crippling disease that affects 200 million people worldwide.

The Ottawa Hospital’s Dr. William Stanford, senior author of the study published in Stem Cells Translational Medicine, had observed that mice with age-related osteoporosis tend to have low levels of healthy mesenchymal stem cells. He theorized that injecting the diseased animals with mesenchymal stem cells from healthy mice should ease the condition. Six months after injecting the mice, the team found that osteoporotic bone had given way to healthy, functional bone.

“We had hoped for a general increase in bone health,” co-author Dr. John E. Davies said in a University of Toronto news release. “But the huge surprise was to find that the exquisite inner ‘coral-like’ architecture of the bone structure of the injected animals — which is severely compromised in osteoporosis — was restored to normal.”

About one-quarter of Canadians will suffer a fracture because of osteoporosis at some point in their lives, costing the health-care system more than $2.3 billion each year. There are few treatment options for the condition.

Mesenchymal stem cells can extracted fairly easily from bone marrow, adipose (fat) tissue, skeletal muscle, umbilical cord blood, placenta, and other sources. A large part of their appeal is they can be transplanted from person to person without being rejected by the body’s immune system. Research also suggests they have anti-inflammatory qualities.

“We stumbled into the bone research field completely by chance a number of years ago, but we felt it was very important to pursue this because age-related osteoporosis takes a huge toll on people and the health-care system,” said Dr. Stanford in an Ottawa Hospital news release. “Obviously we have a lot more work to do, but I’m very excited by the potential that this research could one day help a lot of people.”

These and other intriguing and provocative questions will be addressed at the March 24th Information Day on Cell Therapy at the Ottawa Westin Hotel. Organized by the CellCAN Regenerative Medicine and Cell Therapy Network in partnership with BioCanRx Biotherapeutics for Cancer Treatment, the goal of the event is to separate myth from reality in cell therapy.

The information session, which runs from 10 a.m. to 3 p.m. in the Westin’s Governor General Ballroom, is free and the general public is welcome. Highlight of the day will be a panel discussion from noon to 1:30 p.m. featuring:

Dr. Lauralyn McIntyre, a physician/researcher at the Ottawa Hospital who is investigating how stem cells can improve outcomes in septic shock;

Dr Kelley Parato, the Director of Scientific Affairs at BioCanRx who is working to turn immune cells into weapons to kill cancer cells; and

Dr Bernard Thébaud, a neonatologist at the Children’s Hospital of Eastern Ontario and a senior scientist with the Ottawa Hospital Research Institute whose team is developing stem cell treatments for babies with life-threatening lung diseases.

Information booths will be set up in the ballroom and co-sponsors include the Ottawa Hospital, the Children’s Hospital of Eastern Ontario Research Institute, Canadian Blood Services, the Leukemia & Lymphoma Society of Canada, the University of Ottawa and the Networks of Centres of Excellence of Canada.

Recently we blogged about the proliferation of clinics offering an expensive, unproven stem cell treatment for arthritic and damaged knees.

In the current edition of the Clinical Journal of Sport Medicine, the Australasian College of Sports Physicians (ACSP) published a position paper on the use of mesenchymal stem cell therapies in sports and exercise medicine.…

Recently we blogged about the proliferation of clinics offering an expensive, unproven stem cell treatment for arthritic and damaged knees.

In the current edition of the Clinical Journal of Sport Medicine, the Australasian College of Sports Physicians (ACSP) published a position paper on the use of mesenchymal stem cell therapies in sports and exercise medicine. You can find the full paperhere. It’s well worth reading if you are thinking about spending thousands of dollars for such a treatment or know someone who is.

Mesenchymal stem cells are commonly extracted from adipose (fat) tissue, skeletal muscle, umbilical cord blood, placenta, and bone marrow. In our blog post we dealt mainly with a same-day treatment known as the BMAC (bone marrow aspirate concentrate) procedure. It involves extracting the mesenchymal stem cells from a patient’s pelvis, running them through a centrifuge to concentrate them, and then re-injecting them into the bad knee to reduce inflammation and repair damage. The ACSP, which represents sports physicians in Australia and New Zealand, also considered a treatment in which cells are extracted via liposuction from fat tissue and then put through the centrifuge — a procedure the U.S. Food and Drug Administration is cracking down on.

The paper suggests technology is running ahead of scientific knowledge: “A contemporary development in manufacturing techniques uses far shorter preparation times (often less than two hours) at far less cost, producing a non-expanded mixed cell population. However, mesenchymal stem cell numbers are low in these preparations and the presence of multiple cell types raises questions about the efficacy of these techniques. Although the progress in biotechnology has resulted in commercially attractive outcomes, the science substantiating their effectiveness has lagged behind … No published randomized controlled trials for ‘same-day bone marrow stem cell concentrates’ are available as of 2015.”

This is not to say such treatments don’t work, but that much more study is needed. And it needs to meet the gold standard of a double-blind, randomized, placebo-controlled clinical trial in which some patients get the mesenchymal stem cells and others get a placebo treatment. The ACSP found only one such trial has been done and it did not involve BMAC. Rather, it demonstrated no difference in outcomes between the treatment group who received adipose-derived mesenchymal stem cells and those who got a saline injection. The full study has yet to be published.

According to the ACSP, using stem cells to treat damaged knees should be initiated under only two conditions:

As part of a rigorous clinical research trial.

As an individualized innovative therapy where there is a commitment to the transparent collection of data for analysis by a research body external to the clinic.

“Innovative therapy” refers to a treatment undertaken by a physician in the hope it will help the patient, though its effectiveness has yet to be proven. The upside is that this is how medical practice improves: doctors try something and it works. The downside, as the ACSP notes, is that such treatments may become adopted practices with no proof they actually do any good. The worst case scenario is that an unproven treatment actually does harm.

Randomized, placebo-controlled clinical trials can take years and cost hundreds of thousands of dollars. To someone with arthritic or damaged knees — or hips or shoulders for that matter — it’s cold comfort to hear that more study is needed before such stem cell treatments get the thumbs up or thumbs down. In the meantime, we don’t know.

Nancy Reagan, whose devotion to her husband President Ronald Reagan over their 52 years of marriage was the stuff of legend, is being remembered today as a woman of amazing strength and character.…

Nancy Reagan, whose devotion to her husband President Ronald Reagan over their 52 years of marriage was the stuff of legend, is being remembered today as a woman of amazing strength and character.

Mrs. Reagan, who died Sunday at age 94, not only provided full-time care to her husband for the last 10 years of his life — as Alzheimer’s ravaged his memory — she became a tireless advocate for advancing stem cell science.

Breaking with the conservative element of the Republican Party, she urged George W. Bush’s Administration to reverse its limitations on stem cell research.

As Pam Solo and Gail Pressberg describe in their book The Promise and Politics of Stem Cell Research, Mrs. Reagan hoped that stem cells might provide new treatments for a variety of diseases and conditions, well beyond her husband’s Alzheimer’s: “I just don’t see how we can turn our backs on this.”

Arnold Schwarzenegger, like President Reagan a former governor of the State of California, tweeted that “Nancy Reagan was one of my heroes” and a person of “unbelievable power, class and grace,” who “left her mark on the world.”

Mr. Schwarzenegger was governor when California began investing $3 billion in stem cell R&D to find cures for a number of diseases and health conditions. The California Institute for Regenerative Medicine has since become one of the driving forces in stem cell R&D in the world.

Mrs. Reagan was active with Michael J. Fox in raising millions of dollars to fund scientists’ investigations into the dementia, Parkinson’s and other diseases.

“Is half a billion dollars too big a budget ask to cure chronic conditions such as diabetes, heart disease, multiple sclerosis and Parkinson’s that account for the bulk of Canada’s health care costs?”

The article, available here, profiles the Canadian Stem Cell Foundation’s Pre-Budget Submission to the House of Commons Finance Committee calling on the federal government to commit $250 million in 2016 and an equal amount five years later to support the $1.5 billion Canadian Stem Cell Strategy. …

“Is half a billion dollars too big a budget ask to cure chronic conditions such as diabetes, heart disease, multiple sclerosis and Parkinson’s that account for the bulk of Canada’s health care costs?”

The article, available here, profiles the Canadian Stem Cell Foundation’s Pre-Budget Submission to the House of Commons Finance Committee calling on the federal government to commit $250 million in 2016 and an equal amount five years later to support the $1.5 billion Canadian Stem Cell Strategy. The federal outlay will be doubled with more than $1 billion from other sources, including the private sector, philanthropists, health charities and other partners.

Foundation President & CEO James Price told Re$earch Money that the government has been receptive to the case that he and coalition members have been making for the Strategy in the lead-up to March 22 tabling of Budget 2016. “Our proposal is strongly aligned with what we’re hearing in terms of the government’s proposed innovation agenda looking at investments that target key growth sectors for Canada that have the ability to attract investment, grow competitive export-oriented companies and diversify the economy.”

The article points out that the lion’s share of private funds would come from STEMCELL Technologies Inc., a Vancouver-based firm that plans to invest $350 million to $500 million in R&D over the Strategy’s 10-year time span. “If you want faster results (from stem cell research) you need a coordinated approach across Canada and that requires money,” says Dr. Allen Eaves, STEMCELL’s President and CEO.

Across the United States and in at least one Canadian city, people are paying thousands of dollars for an unproven, same-day stem cell treatment to ease pain and restore flexibility in their arthritic or damaged knees.…

Across the United States and in at least one Canadian city, people are paying thousands of dollars for an unproven, same-day stem cell treatment to ease pain and restore flexibility in their arthritic or damaged knees.

The question is: does it work?

“We don’t have any really good, level one evidence out there yet,” says Dr. Shane Shapiro, an orthopedic surgeon at the Mayo Center for Regenerative Medicine in Jacksonville, Florida. “But judging by the proliferation of practices that perform the procedure and the number of patients that have it, many people are willing to jump in without knowing whether it will or won’t work.”

Dr. Shapiro leads a 25-patient clinical trial investigating whether the bone marrow aspirate concentrate (BMAC) procedure actually relieves pain and helps knees heal in patients with osteoarthritis. Over the past three years he and a team of researchers extracted mesenchymal bone marrow stem cells from patients’ pelvises, spun them in a centrifuge to a concentrated state and then re-injected them into one knee. The other knee, serving as a control for the experiment, got a placebo injection of saline solution. Patients then underwent magnetic resonance imaging (MRI) at six months and a year to see if the knee cartilage improved from its original inflamed state.

Mesenchymal stem cells are used because they can differentiate into bone cells, cartilage cells and muscle cells. They also have anti-inflammatory qualities, which could be helpful in treating osteoarthritis. The process is considered “autologous” because the cells are reinjected into the same person who provided them, as opposed to “allogenic” transplants that involve cells donated from another person.

Dr. Shapiro expects the results to be published soon. Meanwhile, clinics offering the BMAC procedure are popping up all over and charging between $1,800 and $10,000 per injection. ”Unfortunately, now you can take a weekend course or four-day course in how to do this,” says Dr. Shapiro. “I actually don’t think that all places that you can get this done are reputable, but there are some.”

What does the FDA say?

The U.S. Food and Drug Administration (FDA) has published draft guidance documents that reflect the agency’s current thinking about such treatments. At issue is whether the procedure constitutes more than “minimal manipulation” of the cells based on the degree of processing they undergo, and represents “homologous use” — meaning the cells perform their same basic function at the site in which they are injected. The draft guidance seems to indicate the BMAC procedure meets the criteria of minimal manipulation and homologous use,

When we sought clarification, the FDA’s Center for Biologics Evaluation and Research wrote in an email that it “expects anyone involved with human cells, tissues, and cellular and tissue-based products (HCT/Ps), including stem cells, to familiarize themselves with the regulations; they are responsible for self-assessing how the appropriate regulations apply to their products.” It added that the attributes of “processing/manipulation and intended use must be carefully evaluated on a case by case basis.”

Health Canada, which takes a harmonized approach to that of the FDA, has not yet considered whether BMAC meets minimal manipulation or homologous use criteria. According to a statement from the Biologics and Genetic Therapies Directorate, “In some cases, autologous cell therapy products that are processed for a particular patient by a regulated health professional pursuant to the scope of their practice may not require federal pre-market regulatory authorization under the Food and Drug Regulations.”

In 2014 the U.S. Federal Appeals Court ruled in FDA’s favour in a case against Regenerative Sciences, a Colorado-based organization that until 2010 had been offering the BMAC treatment with cells that were extracted, cultured to make them more robust, and then re-injected into the patient. The organization now offers the treatment — called Regenexx-C (for “cultured,” a process that takes several days) — only at a clinic in the Cayman Islands, beyond the FDA’s jurisdiction. They continue to offer Regenexx-SD (for “same day”) at the Centeno Shultz Clinic in Colorado and at a network of clinics around the United States.

At a recent webinar, Dr. Christopher Centeno said that while “there are some additional regulations that may be coming out on stem cell therapies,” he didn’t anticipate that “any of those are going to change what happens here at Regenexx” and added that “right now there are no FDA guidelines for this type of work.”

The BMAC procedure should not be confused with stromal vascular fractioning (SVF) in which adipose (fat) cells are drawn from a patient by liposuction, run through a centrifuge to isolate the stem cells and then re-injected into the patient’s damaged knees or hips or shoulders. In fact, many clinics offer the treatment for a laundry list of conditions and diseases. Because SVF breaks down and eliminates the structural components of the adipose cells, it goes beyond the limits of minimal manipulation, according to the FDA. The agency has begun cracking down on clinics offering the procedure.

Here in Canada, clinics are offering BMAC procedures, says Dr. Jas Chahal, an orthopedic sports medicine surgeon with the Women’s College Hospital and the University Health Network in Toronto. “You can get it done. I’m not sure if you can get it across Canada, but there are people doing it in the Greater Toronto Area, for sure.”

Canadian study goes beyond BMAC

Dr. Chahal says more study is needed to prove whether the BMAC procedure is effective. “If you look at some of the articles presented at conferences, the basic science evidence shows that there could be a benefit — there are some anti-inflammatory molecules in there and there are some growth factors. I just don’t think it’s been adequately studied.”

Dr. Chahal and his team are midway through conducting a clinical trial that goes well beyond the same-day BMAC procedure. They are extracting the mesenchymal bone marrow stem cells from their patients and doing ex-vivo expansion (ramping up the number of cells in a lab) and then re-injecting them at concentrations of 1 million, 10 million and 50 million cells. “We’ve enrolled 12 patients and treated six so far,” says Dr. Chahal. “We expect to treat the next six in the next six months and should have data in another year.”

Should the Mayo Clinic study show positive results, Dr. Shapiro expects it could take some time for the BMAC procedure to become standard treatment for osteoarthritis. For one thing, there’s the cost.

“Someone has to pay for this,” says Dr. Shapiro. “If you’re going to be delivering it to millions of people, it has to come down in cost. It’s got to be more efficient and there’s got to be a system where it’s covered under either public or private health insurance, so that we could deliver it to the large number of people who need it.”

It can take millions of cells to do the most simple stem cell transplant. Coming up with ways to produce huge volumes of pure, safe cells is a challenge.…

It can take millions of cells to do the most simple stem cell transplant. Coming up with ways to produce huge volumes of pure, safe cells is a challenge. In mid-January, Prime Minister Justin Trudeau made an appearance at the MaRS Discovery District building in downtown Toronto to announce $20 million in federal funding for advanced therapeutic cell manufacturing to be managed by the Centre for Commercialization of Regenerative Medicine (CCRM), with GE Healthcare also committing $20 million. CCRM’s CEO Dr. Michael May talks about plans for the centre.

Q: How did this come about?

A: This has been evolving over five years as we developed very strong relationships with industry leaders, of which GE HealthcCare is one. As the industry evolves, manufacturing issues are maybe the most talked about bottleneck in cell and gene therapy.

Q:What is your centre going to do?

A: This centre is a business unit of the overall CCRM operation. It has two main activities. One is targeting particular bottlenecks — strategic gaps in the industry — around particular cell types and specific operations and then inventing solutions that can be commercialized as tools and devices. The second thing is the centre will utilize the expertise in the team and the new solutions that will be invented, or technologies that will be integrated, to tackle company problems on a fee-for-service basis.

Q:Can you give a hypothetical example?

A: Any company, large or small, or an academic who is making cells likely has not addressed scale-up and manufacturing in an appropriate way. They are a potential client for not only the solutions we will come up with but also the fee-for-service optimization and scale-up that we will do.

Q:So, if I’m a scientist and I want to do some testing using a particular cell type, but I need millions and millions of cells, I come to you?

A: Yes. We might help in lots of different ways. If there is scale-up that’s needed, we would advise on what needs to get done and create a project around that. And we could hopefully help fund it. It could be fee-for-service or a co-development or any combination.

Q:The space that you have, will scientists be using it themselves or will they be hiring your team to do something for them?

A: It’s not that we would advise them what to do and they would go off to their labs and do it. This will be an advanced manufacturing centre. Our focus with this funding will be our new Good Manufacturing Practice (GMP) facility to make cells for clinical trials. We are going to hire 30 to 40 people to conduct these projects and do this fee-for-service work.

Q:You’ve got GE Healthcare as a major partner, but do you want more businesses to come in?

A: This is a consortium model. GE Healthcare is an anchor partner but we want to bring together and integrate technologies from other sources as well. So, we’re looking for technology partners. But we’re also looking for clients — cell therapy companies that need these solutions. An advantage of the site is it is in such a rich clinical environment in Toronto and, more broadly, Canada. That was a great attraction for GE because it enables them to engage with their customers in clinical trials and projects where the technologies can be stress-tested. This is real-time engagement of the community and the market.

Q:What’s the facility itself?

A: The facility is in MaRS (a not-for-profit corporation founded in downtown Toronto in 2000). It’s going to be part of an entire floor of activities focused on regenerative medicine. There will be the GMP facility there. Our CCRM employees will be there. It will also be the headquarters for the Ontario Institute for Regenerative Medicine and the University of Toronto’s new Medicine by Design project. So when people enter the 10th floor of MaRS, they are going to see a very integrated, co-ordinated ecosystem in and around cell therapy and regenerative medicine.

Q:Will you be working with other cell manufacturing centres like the one opening in Edmonton, and centres in Laval, Montreal and Ottawa?

A: We’re working with those through CellCAN, that’s one point of contact. I was just in Montreal and promoting the idea of the centre being the process development arm. If we need to optimize or scale up manufacturing we can do it in our centre and transfer those solutions back to manufacturing facilities as needed across the country.

Q:So it’s the Canadian model of working collaboratively, not competitively?

A: Absolutely. Although this will be, within MaRS, a very unique ecosystem, it has to be integrated with other activities across the country. It has to be integrated with other activities across the globe. Trying to achieve the proper scale is impossible with one centre or even one country.

Q: What are the anticipated outcomes?

A: The outcomes will be new technologies and tools to enable clinical- and commercial-scale manufacturing of cells — so there will be very tangible widgets that come out. Over the medium term, there will be a blueprint for cell manufacturing of the future,with integration of a number of technologies. We believe this centre will attract clinical trials to Canada and accelerate the development of technologies and clinical translation so more patients will be receiving cell therapies. It will support companies created in Canada but, as I mentioned, attract companies to Canada. We can’t just talk about scientific leadership anymore. We have to be leading commercialization. We need to be leading translation, through clinical trials. And this piece is manufacturing. Because with manufacturing comes stickiness and companies that are sustainable in Canada.

Q:What do you mean by ‘stickiness?’

A: This is advanced manufacturing; it’s not like making a car that you can diffuse production to the cheapest site in the world. This is an area where the leading edge is still being developed. If we are the leaders in manufacturing cells and those cells get manufactured here, then the companies that are here and the jobs we create here will be sticky.

Federal regulators in the United States are about to crack down on hundreds of clinics peddling pricey stem cell therapies to treat a laundry list of diseases and conditions without scientific evidence to back them up.…

Federal regulators in the United States are about to crack down on hundreds of clinics peddling pricey stem cell therapies to treat a laundry list of diseases and conditions without scientific evidence to back them up.

According to a report by STAT, an online health and medicine news service, some 200 stem cell clinics have cropped up in recent years, selling injections, facelifts, and treatments that have not undergone clinical trials.

Many of the clinics use a procedure called stromal vascular fraction (SVF) in which cells are extracted from a patient by liposuction, run through a centrifuge to collect the adipose (fat) stem cells and returned to the patient intravenously or injected at the site of the condition. Clinics promote the SVF procedure to treat a variety of conditions such as arthritis, Parkinson’s disease, pulmonary fibrosis, chronic obstructive pulmonary disease, multiple sclerosis, cerebral palsy, and amyotrophic lateral sclerosis. Treatments can cost anywhere from $5,000 to $25,000.

The American Food and Drug Administration (FDA) recently issued draft guidelines on the use of human cells, tissues, or cellular or tissue-based products and will hold a public hearing on April 13 at Silver Spring, Maryland to help finalize its stand on such treatments.

As STAT reported, the FDA already sent a warning letter to a network of stem cell clinics in California, New York, and Florida advising the owner that he needed FDA approval to sell and use stem cells, which the agency classified as biological drugs.

FDA approval requires evidence that stem cell treatments are safe and effective, which, as the article points out, “takes drug companies many years of clinical trials to obtain, at a cost of millions of dollars.”

Canadian bioethicist Dr. Leigh Turner of the University of Minnesota has long criticized the FDA for failing to crack down on the clinics as the mushroomed across the U.S. “If it’s not safe and it’s not going to help patients,” Dr. Turner told STAT. “It’s just predatory behavior.”

Young scientists who have done something remarkable in stem cell research recently have until March 1 to tell the world about it and win a $25,000 award.…

Young scientists who have done something remarkable in stem cell research recently have until March 1 to tell the world about it and win a $25,000 award.

Early career scientists — younger than 45 as of Jan. 1 2016 — working in the field of stem cells and/or regenerative medicine are eligible for the annual Stem Cells and Regenerative Medicine Award. Entrants must submit an essay describing the implications of work they have done in the past three years in developing cell-based treatments for cancer, degenerative disorders and immunological conditions.

Sponsored by China-based Boyalife, the largest stem cell bank in Asia, and the American Association for the Advancement of Science, publisher of Science, Science Translational Medicine, Science Signaling and Science Advances, the award recognizes significant contributions in advancing basic science to clinical applications in the field.

The winning essay will be published in Science. The runner-up will receive $5,000. Find out more here.

A “Dream Team” of top Canadian scientists, led by two outstanding figures in stem cell research, is launching a new attack on brain cancers.…

A “Dream Team” of top Canadian scientists, led by two outstanding figures in stem cell research, is launching a new attack on brain cancers.

The team, announced today in Toronto, will receive $11.7 million over four years from Stand Up To Cancer Canada (SU2C), Genome Canada, the Canadian Institutes of Health Research, the Cancer Stem Cell Consortium, and Ontario Institute for Cancer Research.

Dr. Peter Dirks, a neurosurgeon at Toronto’s SickKids Hospital who first identified cancer stem cells in brain tumours, leads the team while Dr. Sam Weiss, director of the University of Calgary’s Hotchkiss Brain Institute is co-leader. A member of the Canadian Stem Cell Foundation’s Science Advisory Council, Dr. Weiss discovered adult neural stem cells.

“Brain tumours in children and adults contain a small number of cells called stem cells that resist treatment and continually regenerate, driving tumour growth and recurrence after initial responses to treatment,” Dr. Dirks said in a news release. “Our team will conduct multiple analyses of brain cancer stem cells, profiling their biological makeup to identify drugs that are likely to block the uncontrolled growth of the tumours, and carry out clinical trials across Canada to find the safest and most effective drugs to treat these cancers.”

Dr. Michael Salter, The Hospital for Sick Children Research Institute; and

Dr. Michael D. Tyers, University of Montreal.

“Brain tumours are not as common as many other forms of cancer, but they are devastating, especially when they strike the very young,” said Dr. Phillip A. Sharp, a Nobel laureate and professor at the Massachusetts Institute of Technology. Dr Sharp, who co-chairs the SU2C Scientific Advisory Committee, said the Dream Team “will bring new insights to brain cancer research, which has been an underfunded area.”

Co-chair Dr. Alan Bernstein, President & CEO of CIFAR (Canadian Institute for Advanced Research), said the Dream Team builds on Canada’s research strengths. “Stem cells and cancer stem cells were discovered in Canada,” said Dr. Bernstein, who also chairs the Canadian Stem Cell Foundation’s Board of Directors. “By bringing together a top-flight team of scientists and clinicians from across Canada and applying what we have learned about brain cancer and cancer stem cells, our hope is that novel treatments will be developed.”

Science Minister Dr. Kirsty Duncan welcomed the new Dream Team: “In supporting the Dream Team, the Government of Canada is investing in promising discoveries that could improve the outcome of patients who live with brain cancer.”

Her sentiments were shared by Health Minister Dr. Jane Philpott: “Canada is a leader in stem cell research, and the Dream Team is carrying on this proud tradition,” said Dr. Jane Philpott, Minister of Health.

Headlined “Canada’s stem cell research needs ‘big investment’ to move forward” it quotes Dr. Janet Rossant, President & Director of the Ontario Institute for Regenerative Medicine, as saying Canada does “extremely well on relatively small investments,” but we are not investing the same percentage in stem cell research and development as some of our competitor countries.

Reporter Joseph Quigley cites figures from the Canadian Institutes of Health Research that show the Government of Canada has invested about $705 million in stem cell research since 2001, including $64.5 million in the 2014-15 fiscal year. In comparison, he notes, California — with a population similar to that of Canada — committed $3 billion in funding in 2004, to be rolled out over about 10 years.

“We’ve been at the forefront, we need to make that next big investment to move the field forward,” says James Price, President & CEO of the Canadian Stem Cell Foundation.

The Canadian Stem Cell Strategy, he points out, is focused on delivering 10 new curative therapies to the clinic in 10 years while producing 12,000 jobs for Canadians.

The stem cell community is mourning the loss of one of its most cherished champions, Andrew David Lyall.

Mr. Lyall — Drew to all who knew him — was the inaugural executive director of the Stem Cell Network and, over 12 years, helped build it into an internationally respected research and development powerhouse.…

The stem cell community is mourning the loss of one of its most cherished champions, Andrew David Lyall.

Mr. Lyall — Drew to all who knew him — was the inaugural executive director of the Stem Cell Network and, over 12 years, helped build it into an internationally respected research and development powerhouse. He died at the Élisabeth Bruyère Hospital in Ottawa on Saturday a few days shy of his 50th birthday after an eight-year battle with melanoma.

“He did more than anyone who wasn’t a scientist to advance the field of stem cells in Canada,” said James Price, President and CEO of the Canadian Stem Cell Foundation, where Mr. Lyall previously served as a director. “He emulated the spirt of collaboration that can be traced back to James Till and Ernest McCulloch and that has been vital to the field’s success over the past 15 years.”

Dr. TilI, the surviving partner of the research duo that proved the existence of stem cells more than 50 years ago, credited Mr. Lyall with persuading stem cell scientists across Canada to integrate their efforts. “Drew played a crucial role in the success of the Network, which built an extraordinary community of researchers and scholars. It also played an important role in the establishment of several other innovative initiatives, such as the Canadian Stem Cell Foundation. The impact will be felt for years to come.”

While dealing with his own imminent death, Mr. Lyall remained a model of calm dignity. He kept friends up to date via a lively and often funny private blog. When his treatment shifted from therapeutic to palliative care he was realistic but unfailingly upbeat. He posted that he had “enjoyed a life to be very grateful for” and encouraged friends to write letters to their own children, as he had done, telling them “what you value about them, the good times you have had, and things you wished you had done together.” An organizer to the end, he used his time in the hospice to create a namesake fund at the Ottawa Regional Cancer Foundation.

Dr. Connie Eaves, a leading stem cell scientist who helped put the Stem Cell Network together, recalled how Mr. Lyall made an indelible impression during his job interview.

“Ron Worton (the Network’s founding Scientific Director) briefed me about Drew just before we were to interview him — me by teleconference — for the top position in the admin group. Drew’s intelligence and thoughtfulness, combined with that irresistible enthusiasm for working in the discovery arena with not too many rules, were captivating. We were so lucky he came on board. The world has lost a very special and good person.”

Mr. Lyall’s influence was felt well beyond Canada’s borders. A founder of the International Consortium of Stem Cell Networks, he worked closely with colleagues around the world, urging them to pool their talents in the service of finding cures and saving lives.

“It was his unique leadership style that allowed all of these different countries to come together and share a joint perspective of what was possible,” said Nancy Witty, CEO of the International Society for Stem Cell Research. “There were few people who could bring his special personality to making all of us friends and colleagues that care about one another.”

That style might best be described as disarmingly casual, relaxed bordering on rumpled. He could step up to a conference podium unfazed that his shirttail was untucked and enthrall an audience with an engaging presentation delivered in a butterscotch brogue that had lost none of its flavour since he relocated to Canada from Scotland in late 1992.

“He was unbelievably intelligent, but you didn’t necessarily realize it right away because he was not the pressed, corporate type,” said Shannon Sethuram, who worked closely with Mr. Lyall at the Stem Cell Network for five years and more recently at BioCanRx, another federally funded research network. “There was something very casual about him that spoke to his confidence in who he was. He was, bar none, the best person I have ever worked with.”

Mr. Lyall was born on Jan. 21, 1966 in Edinburgh. He was the son of David Lyall, a Presbyterian minister, and Margaret Lyall and a brother to Catherine Slatter. He studied politics, philosophy and economics at Oxford but was an accountant by profession. He relocated to Canada to be closer to Suzanne Loch, an Ottawa school teacher he had met while travelling. They married in 1993 and have two children Heather, 15, and Matthew, 12.

“Drew was just passing through Canada when he met Suzanne on a train, and, being totally and understandably charmed, he decided to extend his stay,” said Patrick Lafferty, a mentor to Drew when he came to work at the Ottawa office of Coopers & Lybrand (which later became Pricewaterhouse Coopers — PwC). “Our United Kingdom firm had an enviable model of hiring the best and brightest liberal arts graduates from their leading universities and then teaching them accounting and auditing. Drew would happily accept any challenge as an opportunity, begin thinking it through, come back a few days later, challenge some of the assumptions, and then offer some real insights on how to best pull it off.”

Mr. Lafferty admired Mr. Lyall’s zest for life. “From the beginning, he threw himself into his first baseball game, his first hockey game, every office party, charity fundraising and sporting event, and of course, into his delightful wedding to Suzanne.”

Though not particularly athletic — he played recreational soccer and took skating lessons — Mr. Lyall was an avid sports fan who faithfully followed long-suffering Hibernian FC of North Edinburgh’s Leith. The “Hibs,” he wrote in his blog, “haven’t won the main Scottish cup for 114 years now.” He knew they were not very good, said close friend Randy McIntyre, “but he was quite proud of them.” In Canada, he quickly adopted American football, travelling to Wisconsin to see his beloved Green Bay Packers play and purchase Cheesehead hats for his children. Over the Christmas holidays, he was able to leave the hospice for a morning of watching “footie” with friends over a full English breakfast at an Ottawa pub. He tracked the ups and downs of the Ottawa Senators right up to a few days before his death.

Mr. Lyall travelled extensively, visiting more than 80 countries, but left the world regretting that he was never able to visit Machu Picchu, the Incan citadel of Peru’s Andes Mountains. A voracious reader, he Lyall, could speak as knowledgeably about the popular novels of Nick Hornby as he could debate Clayton M. Christensen’s theories on the impact of disruptive technologies on established industries.

With no science training to speak of, he quickly grasped the subtleties of the complex field he championed. A quiet but persuasive force, Mr. Lyall didn’t feel the need to be regarded as the smartest person in the room. “But he often was,” said Dr. Michael Rudnicki, the Stem Cell Network’s Scientific Director and CEO. “True leaders don’t bully their way forward. They prepare the ground. He was a builder who wanted to do great things.”

Dr. Rudnicki believes Mr. Lyall’s professional legacy is the community of stem cell scientists spread across Canada who agreed to work together collaboratively under the Network banner. “The nature of the community reflected who he was and how he dealt with people.”

Dr. Janet Rossant, President and Executive Director of the Ontario Institute for Regenerative Medicine, agreed. “Drew was a true stem cell hero,” she said. “He was the glue that brought the Network together, whether driving the science, advocating on the Hill for a cord blood stem cell bank, or leading the party at the annual Network pub night.”

Those sentiments were echoed by Prof. Tim Caulfield, Canada Research Chair in Health Law and Policy at the University of Alberta. “A master at bringing people together, Drew did much to help build Canada’s stem cell community. He always seemed to be at its centre.” And by Dr. Sam Weiss, Director of the University of Calgary’s Hotchkiss Brain Institute: “Drew was a warm and wonderful person to know, have a drink with and to share the achievements of collaboration and friendship.”

Mr. Lyall was one of the key people responsible for getting stem cell researchers to embrace the concept of commercializing their work so that it could be carried forward to clinical therapies. Dr. Michael May, President and CEO of the Centre for Commercialization of Regenerative Medicine said his organization, spun out of the Network, wouldn’t exist without Mr. Lyall’s contributions.

“Drew played a critical role in having the academic community embrace us, helping facilitate connections with the academic partners,” said Dr. May. “He helped me, personally, make those founding connections within the Canadian community and abroad too. He was very generous with his time, his insights and his network.”

Mr. Lyall also helped develop CellCAN, the Regenerative Medicine and Cell Therapy Network, created to accelerate progress in cell-based therapies. “Drew and the Stem Cell Network have been a driving force in uniting the centres involved in cell manufacturing and enabling the translation of findings at the bench to patients in need of novel and efficient therapies,” said Dr. Dénis Claude Roy, CellCAN’s CEO. “Drew’s trust and involvement were crucial to generate CellCAN.”

Mr. Lyall, who was also an alumnus of the Stanford Graduate School of Business, left the Stem Cell Network in 2013 to become a director general with the Canadian Institutes of Health Research. However, he was lured away last year when Dr. John Bell launched BioCanRx to advance anti-cancer biotherapeutics.

“Early on, I approached Drew for some quick tips about how best to put together a network proposal,” said Dr. Bell. “I left with a lot more than advice. Drew instilled in me a vision of what a network could be and the value it could bring to cancer patients. Once we were awarded the grant, I knew I needed a great CEO to make it all it could be. In almost no time, Drew was able to bring together a very experienced and motivated team and get BioCanRx launched, which reflected how much people enjoyed working with him. Every day I worked with my friend Drew was fun — he had an awesome sense of humour — and made me confident that BioCanRx will be a success.”

Prof. Bartha Maria Knoppers, Director of the Centre of Genomics and Policy at McGill University, admired Mr. Lyall for “always reaching out beyond borders and disciplines,” and saw him as “a true gentleman whose candour and insight immediately attracted trust and participation.”

Dr. Fabio Rossi, Acting Director of the University of British Columbia’s Biomedical Research Centre, said Mr. Lyall was one of the most charming people he ever worked with. “His infectious optimism and enthusiasm motivated all around him. He truly was the Network’s centre of gravity, and responsible for much of the cohesion that made it a success.”

Dr. Rudnicki, who worked side by side with Mr. Lyall for many years at the Network, said his death marks a tremendous loss not just to his family, friends and colleagues, but for health science. “He had so much more to contribute.”

(Joe Sornberger, Director of Communications Programs for the Canadian Stem Cell Foundation, is the author of Dreams & Due Diligence — Till and McCulloch’s Stem Cell Discovery and Legacy. The quotes included here were collected through interviews and email correspondence.)

Today’s announcement of a new centre for advanced therapeutic cell technologies in Toronto is an investment in health-related technology, says Prime Minister Justin Trudeau.…

Today’s announcement of a new centre for advanced therapeutic cell technologies in Toronto is an investment in health-related technology, says Prime Minister Justin Trudeau.

“The health of Canadians is a priority,” Prime Minister Trudeau said at the announcement. “We believe that supporting this new, world-class facility will have significant benefits for innovative health-related technology in Canada and around the world.

GE Healthcare and the Federal Economic Development Agency for Southern Ontario will invest a combined $40 million in partnership with the Centre for Commercialization of Regenerative Medicine (CCRM) to build the centre in Toronto’s MARs discovery district, which is operated by a not-for-profit corporation to commercialize publicly funded medical research and other technologies.

According to a news release, the centre will speed the development of cell manufacturing technologies to improve patient access to new therapies. Scaling up the quantity of stem cells to the vast numbers required for transplantation while ensuring the purity and quality of those cells is vital to moving the regenerative medicine field forward.

James Price, President and CEO of the Canadian Stem Cell Foundation, attended the Toronto event and said the investment will enhance capacity to implement the Canadian Stem Cell Strategy, the private-public partnership that will deliver up to 10 new curative therapies to the clinic within 10 years.

“It’s key piece to the puzzle,” he said, noting that state of the art cell-manufacturing facilities are operating in Laval, Montreal, and Ottawa and that one will open its doors in Edmonton this summer. “These kinds of developments boost Canada’s overall ability to produce cells and attract clinical trials so that we can begin delivering new treatments to a number of diseases.”

Michael May, President and CEO of CCRM, added: “We have built a strong industry consortium of nearly 50 companies to help drive a collaborative approach to realizing the potential of regenerative medicine. GE Healthcare already plays a leading role in that consortium and the company’s deep knowledge of the bioprocessing industry, combined with its global scale and health care insights, makes it the ideal anchor partner for the new centre.”

Kieran Murphy, CEO of GE Healthcare’s Life Sciences business, says it is “increasingly clear that cell therapies and regenerative medicine will transform health care globally, but successful industrialization is now crucial to widespread adoption. This new centre will enable us to work with cell therapy companies to push beyond existing technical limits and problem-solve.”

When most of us think about using stem cells to cure disease, we picture these building block cells being injected into damaged tissues or organs to help repair and rebuild them.…

When most of us think about using stem cells to cure disease, we picture these building block cells being injected into damaged tissues or organs to help repair and rebuild them.

According to a recent Wall Street Journal article, there are more than 300 clinical trials underway around the world to test stem cells’ ability to treat diseases such as heart attack (read about a Canadian study here), stroke and amyotrophic lateral sclerosis — among others. The general idea is to get the stem cells to where the disease is doing damage so they can affect healing.

But, as the WSJ article points out, there is another approach in which stem cells are used to create models of disease outside the human body, in a Petri dish. These models not only provide researchers with a closer look at the molecular makeup of a disease, they offer the opportunity to test drugs that might be effective against it. The article highlights research at the University of California, San Diego where stem cells are being used to make Alzheimer’s neurons to test the safety and effectiveness of potential drug therapies.

There are real benefits to this approach. For one, it’s a supplement to using animals as disease models. Sometimes animals aren’t susceptible to the same diseases as people. In Alzheimer’s, for example, researchers use mice that have been altered to carry different genes or combinations of genes associated with the dementia.

The stem cell model approach also can save time and money. If scientists can rule out a compound as ineffective before moving to clinical trials, it can prevent years of work being done and millions of dollars being spent to travel down a research road that ultimately is a dead end.

Also, advances in technology mean scientists can use high-throughput screening to test hundreds, even thousands, of compounds against model tissue or organs to find a good candidate to shut down the disease — the research equivalent to finding a therapeutic needle in a haystack.